Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, ASGT Executive Office

Size: px

Start display at page:

Download "Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, ASGT Executive Office"

Milton Johnston
5 years ago
Views:

2 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Table of Contents A Welcome from Theodore Friedmann, MD ASGT Officers and Board of Directors Committee Meeting Schedule Committee Rosters Planning Committee and Group Coordinating Reviewers Annual Meeting Supporters Annual Meeting Exhibitors Abstract Reviewers Excellence in Research Award and Travel Grant Recipients Outstanding New Investigator Awards Faculty Listing General Meeting Information Washington State Convention & Trade Center Floor Plans Schedule-at-a-Glance Detailed Program Schedule Exhibit Hall Floor Plan Exhibitor Descriptions Exhibitor Information Seattle Fun Facts Housing Information and Map of Seattle Abstract Directory Abstract Author Index ASGT Executive Office 555 East Wells Street Suite 1100 Milwaukee, WI USA phone fax info@asgt.org

3 Dear Colleagues, On behalf of the American Society of Gene Therapy, welcome to the ASGT 10 th Annual Meeting in Seattle, Washington. The ASGT Program Committee, the many scientific and standing committees, and the abstract review groups have put together a fine meeting that focuses on the striking scientific and clinical advances in our field during the past decade. Included in the program are 19 state-of-the-art scientific symposia, 9 education sessions, 40 oral abstract sessions, 3 poster sessions, meetthe-investigator sessions; and two pre-meeting sessions (featuring an international update and a Bioprocessing session). In addition, there will be a special session presented by the Office of Biotechnology Activities related to the NIH system of oversight of Human Gene Transfer. Some of the subjects that are being presented in depth include stem cell biology, gene-based vaccines, immunology of gene therapy, clinical trials, tissue engineering, infectious diseases, musculo-skeletal disorders, neural disorders, respiratory tract disorders and non-viral and viral gene transfer. Several major highlights of the meeting include the George Stam keynote address by Judah Folkman, M.D. and the Presidential lecture by Cynthia Kenyon, PhD. We will also present a special 10th anniversary symposium on June 2, 2007 that will be devoted to a review of the advances in the field of gene therapy and a presentation of important challenges, recent achievements, new concepts and future directions for basic research and clinical applications in gene therapy. Symposium speakers will include Ernest Beutler, MD of The Scripps Research Institute, Malcolm Brenner, MD, PhD of Baylor College of Medicine, Maria Grazia Roncarolo, MD, PhD of the San Raffaele Telethon Institute for Gene Therapy in Milan and David Baltimore, PhD, past-president of Caltech. The symposium will certainly be one of the memorable highlights of what promises to be an outstanding meeting. I would like to thank our exhibitors and industry supporters for their participation and generosity, in addition to the exceptional faculty who share their knowledge, expertise and time. Also, my sincere gratitude to all of you who helped to develop and organize this wonderful program. And finally, I want to thank all of the Annual Meeting participants who will make this a truly vibrant meeting. Welcome to Seattle. Have a great meeting! Sincerely, Theodore Friedmann, MD President ASGT Officers and Board of Directors President (06/07) Theodore Friedmann, MD University of California San Diego School of Medicine President-Elect (06/07) Arthur W. Nienhuis, MD St. Jude Children s Research Hospital Vice President (06/07) David Bodine, PhD National Institutes of Health, Hematopoiesis Section Secretary (04/07) Kenneth Cornetta, MD Indiana University Treasurer (05/08) Beverly L. Davidson, PhD University of Iowa College of Medicine Board Members Jean Bennett, MD, PhD (07) Xandra Breakefield, PhD (09) Barrie Carter, PhD (08) Luigi Naldini, MD, PhD (08) Katherine Ponder, MD (08) Paul D. Robbins, PhD (07) David W. Russell, MD, PhD (08) Michel Sadelain, MD, PhD (07) Adrian Thrasher, MD, PhD (09) Malcolm K. Brenner, MD, PhD (07) Chair, Advisory Council David A. Williams, MD (09) Editor-in-Chief, Molecular Therapy ASGT Executive Office 555 East Wells Street, Suite 1100 Milwaukee, WI USA Phone: Fax: info@asgt.org Executive Director Elizabeth Dooley, CAE, CMP Membership Coordinator Mary Dean Program Manager Ken Janowski Senior Meetings Manager Nadine Kinnunen Consulting Partner Kay Whalen, CAE Additional On-Site Staff Meghan Arnold Audrey Mudek Katie Muellenbach Paul Rushizky

4 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Committee Meetings Committee terms commence and conclude in June at the Annual Meeting. Incoming Chair and committee member terms officially go into effect on June 2, 2007 at the Business Meeting. All meetings will be held at the Washington State Convention & Trade Center unless otherwise noted. Thursday, May 31 11:30am 12:45 pm 2008 Program Committee Meeting and Scientific Committee Meetings (Room 4C4) 11:30am 12:45 pm Publications Committee Meeting (Room 214) 5:15pm 6:15 pm Education Committee Meeting (Room 214) 5:30pm 6:30 pm Friday, June 1 Public Education & Outreach Committee Meeting (Hyatt-Douglas Board Room) 7:00 am 8:00 am Ethics Committee Meeting (Room 213) 7:00 am 8:00 am Industrial Liasion Committee Meeting (Room 214) 12:15 pm 1:15 pm Advisory Council Meeting (Room 213) 12:15 pm 1:15 pm International Committee Meeting (Room 214) 5:00 pm 7:00 pm Molecular Therapy Editorial Board Meeting and Reception (Grand Hyatt-Steven s Room) 5:15 pm 7:15 pm Clinical & Regulatory Committee Meeting (Room 213) Saturday, June 2 Society Annual Business Meeting The ASGT Annual Business Meeting will be conducted in Ballroom ABC, on Saturday, June 2, 7:15 am 8:00 am. A continental breakfast will be served at 7:00 am. All ASGT members in good standing are welcome to attend. Election results will be announced, new officers will be installed and the Society s finances and membership statistics will be reviewed. We welcome your input and participation! Committee Roster Advisory Council Chair Malcolm K. Brenner, MD, PhD ( ) Member Malcolm K. Brenner, MD, PhD ( ) Theodore Friedmann, MD ( ) Joseph C. Glorioso, PhD ( ) Katherine High, MD ( ) Leaf Huang, PhD ( ) Mark A. Kay, MD, PhD ( ) Donald B. Kohn, MD ( ) Margaret A. Liu, MD ( ) John J. Rossi, PhD ( ) R. Jude Samulski, PhD ( ) Alan E. Smith, PhD ( ) Clinical and Regulatory Affairs Chair Dale Ando, MD ( ) Members Dale Ando, MD ( ) Arthur L. Beaudet, MD ( ) Barry J. Byrne, MD, PhD ( ) Joy Cavagnaro, PhD, DABT, RAC ( ) Kenneth Cornetta, MD ( ) Carl H. June, MD ( ) Andra Miller, PhD ( ) Glenn F. Pierce, MD, PhD ( ) Stephanie Simek, PhD ( ) Kenneth Weinberg, MD ( ) Education Co-Chairs Matthew Weitzman, PhD ( ) Philip L. Leopold, PhD ( ) Roberto Cattaneo, PhD ( ) Members Estuardo Aguilar-Cordova, PhD ( ) Michael A. Barry, PhD ( ) Roberto Cattaneo, PhD ( ) Jay A. Chiorini, MD ( ) Olivier Danos, PhD ( ) Dongsheng Duan, PhD ( ) William Goins, PhD ( ) Terry Hermiston, PhD ( ) Roland W. Herzog, PhD ( ) Karin Jooss, PhD ( ) Tal Kafri, PhD ( ) Donald B. Kohn, MD ( ) Philip L. Leopold, PhD ( ) Fulvio Mavilio, PhD, Prof ( ) Paul B. McCray, Jr., MD ( ) Robin J. Parks, PhD ( ) Joseph Zabner, MD ( )

5 Committee Roster Ethics Chair Dieter Gruenert, PhD ( ) Members Marina Cavazzana-Calvo, MD, PhD ( ) Lawrence C. Chan, MD ( ) Jose B. Cibelli, DVM, PhD ( ) Cynthia Dunbar, MD ( ) Henry T. Greely, JD ( ) Barbara R. Jasny, PhD ( ) Jonathan Kimmelman, PhD ( ) Bernard Lo, MD ( ) Punam Malik, MD ( ) R. Scott McIvor, PhD ( ) Nicholas Muzyczka, PhD ( ) Diane Wara ( ) Neil Weisfeld, JD ( ) Industrial Liaison Chair Douglas J. Jolly, PhD ( ) Members Boro Dropulic, PhD ( ) Mitchell Finer, PhD ( ) Michael Fons, PhD ( ) Anthony Green, PhD ( ) Douglas J. Jolly, PhD ( ) Karen Kozarsky, PhD ( ) Edward Lanphier ( ) Andy Lin, PhD ( ) Gerard McGarrity, PhD ( ) Ralph W. Paul, PhD ( ) Alain Rolland, PharmD, PhD ( ) Vladimir Slepushkin, MD, PhD ( ) Kenneth G. Surowitz, PhD ( ) Geoff Symonds, PhD ( ) Thomas Tillett ( ) Gabor Veres, PhD ( ) Samuel C. Wadsworth, PhD ( ) International Chair Jesus Prieto, MD, PhD ( ) Members Olivier Danos, PhD ( ) George Dickson, PhD ( ) Yoshikatsu Eto, MD, PhD ( ) Noriyuki Kasahara, MD, PhD ( ) Mark A. Kay, MD, PhD ( ) Philippe Moullier, PhD ( ) Jesus Prieto, MD, PhD ( ) John Rasko, MD, PhD ( ) Bruno Sangro, MD, PhD ( ) Sander van Deventer, MD, PhD ( ) Seppo Yla-Herttuala, MD, PhD ( ) Membership Chair Seng H. Cheng, PhD ( ) Members C. Anthony Blau, MD ( ) Seng H. Cheng, PhD ( ) Yoshikatsu Eto, MD, PhD ( ) Donald B. Kohn, MD ( ) Dexi Liu, PhD ( ) Isabelle Riviere, PhD ( ) Paul D. Robbins, PhD ( ) Maria Grazia Roncarolo, MD, PhD ( ) John J. Rossi, PhD ( ) Savio L.C. Woo, PhD ( ) Robert M. Frederickson, PhD ( ) Nominating Chair Donald B. Kohn, MD ( ) Members Xandra Breakefield, PhD ( ) Barrie J. Carter, PhD ( ) Donald B. Kohn, MD ( ) R. Scott McIvor, PhD ( ) Seppo Yla-Herttuala, MD, PhD ( ) Program Co-Chairs Katherine Ponder, MD ( ) Paul B. McCray Jr., MD ( ) Brian Sorrentino, MD ( ) Members Steven M. Albelda, MD ( ) Eric W.F.W. Alton, MD, FRCP ( ) Barry J. Byrne, MD, PhD ( ) Kenneth Cornetta, MD ( ) Ronald G. Crystal, MD ( ) Steven C. Ghivizzani, PhD ( ) Roland W. Herzog, PhD ( ) Philip R. Johnson, MD ( ) Mark A. Kay, MD, PhD ( ) Brendan Lee, MD, PhD ( ) Douglas Losordo, MD ( ) Luigi Naldini, MD, PhD ( ) Matthew H. Porteus, MD, PhD ( ) Darwin J. Prockop, MD, PhD ( ) Michel Sadelain, MD, PhD ( ) Francis C. Szoka, Jr., PhD ( ) Alice F. Tarantal, PhD ( ) David B. Weiner, PhD ( ) Matthew Weitzman, PhD ( ) John H. Wolfe, VMD, PhD ( ) Jon A. Wolff, MD ( ) Joseph Zabner, MD ( ) Publications Chair George Stamatoyannopoulos, MD ( ) Members David Bodine, PhD ( ) Barrie J. Carter, PhD ( ) Katherine A. High, MD ( ) Mark A. Kay, MD, PhD ( ) Hans-Peter Kiem, MD ( ) Donald B. Kohn, MD ( ) Brendan Lee, MD, PhD ( ) Michel Sadelain, MD, PhD ( ) Brian P. Sorrentino, MD ( )

6 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Committee Roster Cancer Gene Therapy Chair Steven M. Albelda, MD ( ) Members Steven M. Albelda, MD ( ) Laurence JN Cooper, MD, PhD ( ) Paul B. Fisher, MPH, PhD ( ) Stephen Gottschalk, MD ( ) John G. Hay, MD ( ) Kristen Hege, MD ( ) Akseli E. Hemminki, MD, PhD ( ) Helen E. Heslop, MD ( ) Dennis D. Hickstein, MD ( ) Andre Lieber, MD, PhD ( ) John C. Morris, MD ( ) James S. Norris, PhD ( ) Daniel H. Sterman, MD ( ) Richard G. Vile, PhD ( ) Masato Yamamoto, MD, PhD ( ) Cardiovascular Gene Therapy Chair Douglas Losordo, MD ( ) Members Andrew H. Baker, PhD ( ) Kenneth R. Chien, MD, PhD ( ) David A. Dean, PhD ( ) David A. Dichek, MD ( ) J. Kevin Donahue, MD ( ) Frank J. Giordano, MD ( ) Roger J. Hajjar, MD ( ) H. Kirk Hammond, MD ( ) William Li, MD ( ) Douglas Losordo, MD ( ) Kieth L. March, MD, PhD ( ) Marc Penn, MD, PhD ( ) Anthony Rosenzweig, MD ( ) Sonia I. Skarlatos, PhD ( ) Matthew L. Springer, PhD ( ) Edith Tzeng, MD ( ) Embryonic/Somatic Stem Cell and Tissue Engineering Committee Chair Darwin J. Prockop, MD, PhD ( ) Members Michael Andreeff, MD, PhD ( ) David T. Curiel, MD, PhD ( ) Ira J. Fox, MD ( ) Linda G. Griffith, PhD ( ) Diane S. Krause, MD, PhD ( ) Michael R. Linden, PhD ( ) Carolyn Lutzko, PhD ( ) Kazue Ohashi, MD, PhD ( ) Darwin J. Prockop, MD, PhD ( ) Evan Snyder, MD, PhD ( ) Gene Therapy of Genetic Diseases Chair Brendan Lee, MD, PhD ( ) Members Andrea Amalfitano, DO, PhD ( ) Valder Arruda, MD, PhD ( ) Fatima Bosch, PhD ( ) Barry J. Byrne, MD, PhD ( ) Fabio Candotti, MD ( ) Seng H. Cheng, PhD ( ) Jim Hu, PhD ( ) Stefan Karlsson, MD, PhD ( ) Brendan Lee, MD, PhD ( ) Carol H. Miao, PhD ( ) Amit Nathwani, MD, PhD ( ) David Schowalter, MD, PhD ( ) Joseph Zabner, MD ( ) Genetic Vaccines Chair Carl H. June, MD ( ) David B. Weiner, PhD ( ) Members Ronald Alvarez, MD ( ) Scott Antonia, MD, PhD ( ) K. Reed Clark, PhD ( ) Stephen L. Eck, MD, PhD ( ) Mary Lynne Hedley, PhD ( ) Helen E. Heslop, MD ( ) Carl H. June, MD ( ) Art Krieg, MD ( ) Thierry C. Vandendriessche, PhD ( ) David B. Weiner, PhD ( ) Hemopoietic Cell Gene Therapy Chair Alice F. Tarantal, PhD ( ) Members Fabio Candotti, MD ( ) Gay Crooks, MD ( ) David W. Emery, PhD ( ) Karin Gaensler, MD ( ) Robert G. Hawley, PhD ( ) R. Keith Humphries, MD, PhD ( ) Punam Malik, MD ( ) Jan Nolta, PhD ( ) Derek Persons, MD, PhD ( ) Karen E. Pollok, PhD ( ) John J. Rossi, PhD ( ) Mark S. Sands, PhD ( ) Alice F. Tarantal, PhD ( ) Christof von Kalle, MD ( ) Kenneth Weinberg, MD ( ) Immunology of Gene Therapy Chair Roland W. Herzog, PhD ( ) Members Bruce R. Blazar, MD ( ) Youhai H. Chen, MD, PhD ( ) Soosan Ghazizadeh, PhD ( ) Roland W. Herzog, PhD ( ) John Iacomini, PhD ( ) Philip R. Johnson, MD ( ) Dwight Koeberl, MD, PhD ( ) Margaret A. Liu, MD ( ) Pedro Lowenstein, MD, PhD ( ) John D. Mountz, MD, PhD ( ) Daniel A. Muruve, MD ( ) Stanley Riddell, MD ( ) Maria Grazia Roncarolo, MD, PhD ( ) Adrian J. Thrasher, MD, PhD ( )

7 Committee Roster Infectious Disease Chair John A. Zaia, MD ( ) Ronald G. Crystal, MD ( ) Members Julie L. Boyer, PhD ( ) Roberto Cattaneo, PhD ( ) Ronald G. Crystal, MD ( ) Boro Dropulic, PhD ( ) J. Victor Garcia-Martinez, PhD ( ) William Goins, PhD ( ) Michael J. Imperiale, PhD ( ) Tal Kafri, PhD ( ) David Klatzmann, MD, PhD ( ) Alexander Kolykhalov, PhD ( ) Bruce L. Levine, PhD ( ) Wayne A. Marasco, MD, PhD ( ) Jeffrey Ulmer, PhD ( ) James M. Wilson, MD, PhD ( ) Stefan Worgall, MD, PhD ( ) John A. Zaia, MD ( ) Musculo-Skeletal Disorder Gene Therapy Chair Steven C. Ghivizzani, PhD ( ) Members Jeffrey Chamberlain, PhD ( ) Paula Clemens, MD ( ) Alan R. Davis, PhD ( ) George Dickson, PhD ( ) Dongsheng Duan, PhD ( ) Christopher Evans, PhD ( ) Dan Gazit, DMD, PhD ( ) Steven C. Ghivizzani, PhD ( ) Stephen D. Hauschka, PhD ( ) Johnny Huard, PhD ( ) George Karpati, MD ( ) Alan J. Nixon, BVSc, MS ( ) Terence A. Partridge, PhD ( ) Michael Themis, PhD ( ) Jon A. Wolff, MD ( ) Xiao Xiao, PhD ( ) Neural Disorders Gene Therapy Chair Beverly L. Davidson, PhD ( ) John H. Wolfe, VMD, PhD ( ) Members Krys Bankiewicz, MD, PhD ( ) Xandra Breakefield, PhD ( ) Mary Bartlett Bunge, PhD ( ) Maria G. Castro, PhD ( ) Nicole Deglon, PhD ( ) John Forsayeth, PhD ( ) Jeffrey H. Kordower, PhD ( ) John H. Wolfe, VMD, PhD ( ) David C. Yeomans, PhD ( ) Nonviral Gene Transfer Vectors Chair Dexi Liu, PhD ( ) Francis C. Szoka, Jr., PhD ( ) Members Roger C. Adami, PhD ( ) Michael A. Barry, PhD ( ) Michele P. Calos, PhD ( ) Mark E. Davis, PhD ( ) Pamela Davis, MD, PhD ( ) Todd Giorgio, PhD ( ) Hideyoshi Harashima, PhD ( ) James G. Hecker, MD, PhD ( ) Jeffrey Hughes, PhD ( ) Kam W. Leong, PhD ( ) Shulin Li, PhD ( ) Dexi Liu, PhD ( ) Ian MacLachlan, PhD ( ) Ram I. Mahato, PhD ( ) Andrew D. Miller, PhD ( ) Yong Serk Park, PhD ( ) Suzie H. Pun, PhD ( ) David A. Putnam, PhD ( ) Theresa M. Reineke, PhD ( ) Daniel Scherman, PhD ( ) Francis C. Szoka, Jr., PhD ( ) Ernst Wagner, PhD ( ) Oligonucleotide Based Therapies Chair John J. Rossi, PhD ( ) Mark A. Kay, MD, PhD ( ) Members Beverly L. Davidson, PhD ( ) Alan Gewirtz, MD ( ) Peter Glazer, MD, PhD ( ) Dieter Gruenert, PhD ( ) Mark A. Kay, MD, PhD ( ) Ryszard Kole, PhD ( ) Anton P. McCaffrey, PhD ( ) Giuseppe Novelli, PhD ( ) Matthew H. Porteus, MD, PhD ( ) John J. Rossi, PhD ( ) Cy A. Stein, MD, PhD ( ) Bruce Sullenger, PhD ( ) Jon A. Wolff, MD ( ) Respiratory Tract Gene Therapy Chair Paul B. McCray, Jr., MD ( ) Eric W.F.W. Alton, MD, FRCP ( ) Members Eric W.F.W. Alton, MD, FRCP ( ) Bruce Bunnell, PhD ( ) Pamela Davis, MD, PhD ( ) David A. Dean, PhD ( ) John F. Engelhardt, PhD ( ) Jim Hu, PhD ( ) Stephen Hyde, PhD ( ) Larry G. Johnson, MD ( ) Paul B. McCray, Jr., MD ( ) Guoshun Wang, DVM, PhD ( ) Daniel Weiss, MD, PhD ( )

8 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Committee Roster Viral Gene Transfer Vectors Chair Brian P. Sorrentino, MD ( ) Barry J. Byrne, MD, PhD ( ) Members Christopher Baum, MD ( ) Frederic D. Bushman, PhD ( ) Barry J. Byrne, MD, PhD ( ) Francois-Loic Cosset ( ) Anja Ehrhardt, PhD ( ) Jeffrey A. Engler, PhD ( ) Howard J. Federoff, MD, PhD ( ) John T. Gray, PhD ( ) Noriyuki Kasahara, MD, PhD ( ) Michael R. Linden, PhD ( ) Hiroyuki Mizuguchi, PhD ( ) Hiroyuki Nakai, MD, PhD ( ) Philip Ng, PhD ( ) R. Jude Samulski, PhD ( ) Brian P. Sorrentino, MD ( ) Richard O. Snyder, PhD ( ) Arun Srivastava, PhD ( ) James M. Wilson, MD, PhD ( ) ASGT Planning Committee & Group Coordinating Reviewers Kenneth Cornetta, MD Indiana University Indianapolis, IN Theodore Friedmann, MD University of California San Diego School of Medicine San Diego, CA Richard Heller, PhD University of South Florida College of Medicine Tampa, FL Paul B. McCray, Jr., MD University of Iowa College of Medicine Iowa City, IA Arthur W. Nienhuis, MD St. Jude Children s Research Hospital Memphis, TN Katherine Ponder, MD Washington University School of Medicine St. Louis, MO R. Jude Samulski, PhD University of North Carolina at Chapel Hill Chapel Hill, NC

ASGT 10 th Annual Meeting Supporters as of May 1, 2007 Partner Abstracts-on-Disc TM Abstract Supplement Printing

4-FYO7-556 General Meeting Support 10 th Anniversary Symposium 10 th Anniversary Symposium 10 th Anniversary Symposium

Support Two Travel Grants Excellence in Research Award Water Bottles Excellence in Research Award Excellence in Research

9 ASGT 10 th Annual Meeting Supporters as of May 1, 2007 Partner Abstracts-on-Disc TM Abstract Supplement Printing Corporate Symposium Contributor Supported in Part by March of Dimes Birth Defects Foundation Grant No. 4-FYO7-556 General Meeting Support 10 th Anniversary Symposium 10 th Anniversary Symposium 10 th Anniversary Symposium Patron General Meeting support Travel Grant Two Excellence in Research Awards General Meeting Support General Meeting Support Two Travel Grants Excellence in Research Award Water Bottles Excellence in Research Award Excellence in Research Award Two Excellence in Research Awards General Meeting Support 5 Internet Stations Exhibit Hall Pocket Guide Partial Support of the International Symposium

Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Nonviral Vectors: 20 Year s Progress toward Gene Therapy ASGT 10 th Annual Meeting Exhibitors as of May 1, 2007 ASGT is

10 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Nonviral Vectors: 20 Year s Progress toward Gene Therapy ASGT 10 th Annual Meeting Exhibitors as of May 1, 2007 ASGT is pleased to have the following companies, organizations and institutions exhibiting at the 10th Annual Meeting! Aldervron, LLC amaxa GmbH American Society of Gene Therapy Amnis Corporation Bentham Science Publishers Ltd. BioLife Solutions, Inc. BioSpherix, Ltd. Boehringer Ingelheim BTX/Harvard Apparatus CellGenix GrmbH Center for Molecular Therapeutics, The Children s Hospital of Philadelphia Cogenics Corning, Inc. Dataworks Development, Inc. DNAVEC Corporation EUFETS AG FightSMA Geneart, Inc. Greater Boston Convention and Visitors Bureau Ichor Medical Systems, Inc. Introgen Therapeutics, Inc. Kinovate Life Sciences, Inc. Lentigen Corporation Mary Ann Liebert, Inc. Millipore Corporation Molecular Medicine BioServices, Inc. Napa Jenomics Corporation National Gene Vector Laboratories Nature Publishing Group Nature Technology Corporation NIH, Office of Biotechnology Activities Northern Lipids, Inc. Nova Matrix/FMC BioPolymer Penn Vector PolyPlus Transfection Puresyn, Inc. Rich-Mar Sartorius Biotech, Inc. SeqWright, Inc. Southern Research Institute Spectrum Laboratories St. Jude Children s Research Hospital Vandalia Research Inc. Vector Gene Technology Company Ltd. Visualsonics Waisman Clinical Biomanufacturing Facility Make time each day to peruse the products, services and publications offered by the exhibiting companies. Stop by the ASGT Booth to pick up your membership pin and abstract CD. Network with and meet new colleagues in a relaxed atmosphere. Stop by the Job Exchange Area.

11 Abstract Reviewers Committee Chair Kenneth Cornetta, MD RNA Virus Vectors Coordinating Reviewer Cynthia Dunbar, MD Reviewers David W. Emery, PhD Stephanie Laufs, PhD Carolyn Lutzko, PhD Bruce E. Torbett, PhD, MSPH AAV Vectors Coordinating Reviewer Jay A. Chiorini, PhD Reviewers Dawn Bowles, PhD Dongsheng Duan, PhD Douglas M. McCarty, PhD Hiroaki Mizukami, MD, PhD Adenovirus Vectors Coordinating Reviewer Andrew Byrnes, PhD Reviewers Michael A. Barry, PhD Lali Medina-Kauwe, PhD Philip Ng, PhD Victor W. van Beusechem, PhD Other DNA Viruses Coordinating Reviewer Samuel D. Rabkin, PhD Reviewers Timothy P. Cripe, MD, PhD Paola Grandi, PhD Yoshinaga Saeki, MD, PhD Feng Yao, PhD DNA Vectorology Coordinating Reviewer Michele P. Calos, PhD Reviewers R. Scott McIvor, PhD Muna Naash, PhD Nancy Smyth Templeton, PhD Physical Methods of Delivery Coordinating Reviewer Richard Heller, PhD Reviewers Ruxandra Draghia-Akli, MD, PhD James E. Hagstrom, PhD Jeffrey Hughes, PharmD, PhD Carol H. Miao, PhD Chemical and Molecular Conjugates Coordinating Reviewer Suzie H. Pun, PhD Reviewers Hai-Quan Mao, PhD David Oupicky, PhD Kevin G. Rice, PhD Lonnie D. Shea, PhD Inborn Errors of Metabolism Coordinating Reviewer Mark Haskins, VMD, PhD Reviewers N. Matthew Ellinwood, DVM, PhD Philip Laipis, PhD Calogera Simonaro, PhD Allesandra Tessitore, PhD Cardiovascular Coordinating Reviewer Timothy O Brien, MD, PhD Reviewers Andrew H. Baker, PhD Barry J. Byrne, MD, PhD Walter J. Koch, PhD Seppo Yla-Herttuala, MD, PhD Neurologic Coordinating Reviewer Krys Bankiewicz, MD, PhD Reviewers Howard J. Federoff, MD, PhD Michael Kaplitt, MD, PhD Jeffrey H. Kordower, PhD Marco Passini, PhD Muscle and Connective Tissue Coordinating Reviewer Jacques P. Tremblay, PhD Reviewers George Dickson, PhD Yves Fromes, MD, PhD Stephen D. Hauschka, PhD Johnny Huard, PhD Infectious Diseases and Vaccines Coordinating Reviewer David Strayer, MD, PhD Reviewers Anton P. McCaffrey, MD, PhD Jayanta Roy-Chowdhury, MD David B. Weiner, PhD John A. Zaia, MD 10 Cancer Immunotherapy Coordinating Reviewer Richard P. Junghans, MD, PhD Reviewers Mark E. Dudley, PhD Alex Tong, PhD Christopher E. Touloukian, MD Cancer Apoptosis and Suicide Coordinating Reviewer Lily Wu, MD, PhD Reviewers Albert Deisseroth, MD, PhD James S. Norris, PhD Ronald Rodriguez, MD, PhD Donna Shewach, PhD Cancer Targeted Gene Therapy Coordinating Reviewer Jack A. Roth, MD, FACS Reviewers Sunil Chada, PhD John Dong, MD, PhD Helen E. Heslop, MD Noriyuki Kasahara, MD, PhD Hematologic Coordinating Reviewer John Rasko, MD, PhD Reviewers Gay Crooks, MD Robert G. Hawley, PhD Dennis D. Hickstein, MD Derek Persons, MD, PhD Lung and Respiratory Disease Coordinating Reviewer David A. Dean, PhD Reviewers Deborah Gill, PhD Uta Griesenbach, PhD Guoshun Wang, DVM, PhD Daniel Weiss, MD, PhD Vector and Transgene Immunology Coordinating Reviewer R. Jude Samulski, PhD Reviewers Guanping Gao, PhD Katherine A. High, MD Paola Leone, PhD Paul D. Robbins, PhD James M. Wilson, MD, PhD Gene Regulation Coordinating Reviewer Punam Malik, MD Reviewers Mirit I. Aladjem, PhD Christopher Baum, MD James Ellis, PhD Adrian J. Thrasher, MD, PhD Oligonucleotide Therapies Coordinating Reviewer Peter Glazer, MD, PhD Reviewers Dieter Gruenert, PhD Matthew H. Porteus, MD, PhD John J. Rossi, PhD Bruce A. Sullenger, PhD Stem Cells Coordinating Reviewer Reviewers Meri T. Firpo, MD Markus Grompe, MD Diane Krause, MD, PhD Mariusz Z. Ratajczak, MD, PhD Cell Processing and Vector Production Coordinating Reviewer J. Fraser Wright, PhD Reviewers John T. Gray, PhD Bruce L. Levine, PhD Maritza C. McIntyre, PhD Isabelle Riviere, PhD Special thanks to the abstract reviewers for their time and expertise.

12 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Excellence in Research Awards For Students and Postdoctoral Fellows Hunter Chen - Student Quantitative Analysis of Intracellular Unpacking of Polymeric DNA Nanoparticles Constructed from Quantum Dot-FRET Jarrod Johnson - Student AAV Exploits Conditions of Cellular Stress Michael Seiler - Postdoctoral Fellow Adoptive Therapy of Tolerogenic Dendritic Cells Mediates Factor VIII Persistence in Hemophilia A Mice Takeshi Suda - Postdoctoral Fellow Development of Computer-Controlled Injection Device for Hydrodynamic Gene Delivery Frederic Vigant - Student Alteration of Adenoviral Liver Tropism Following Hexon Protein Modification Simon Waddington - Postdoctoral Fellow An In Vivo Model To Evaluate Hepatic Targeting of Adenovirus by Coagulation Factor X Jinhui Wang - Postdoctoral Fellow Loss of Newly Formed Double-Stranded Genomes Is the Primary Cause of raav Low Transduction Efficacy Travel Grants For Students and Postdoctoral Fellows Cynthia Bartholomae Kenneth Greenberg Xiucui Ma Kerstin Schwarzwaelder Brian Brown Dirk Grimm Yonatan Mahller Anurag Sharma Suzanne Buckley Olivia Hibbitt Molly Martin Jeffrey Smith M. Candolfi Tomoyasu Higashimoto Jodi McBride Devika Soundara Manickam Alessia Capotondo David Hokey Disha Mody Masataka Suzuki Cassia Cearley Lucienne Ide Eugenio Montini Luk Vandenberghe Yvonne Chen Guiandre Joseph Hiroshi Nakashima Kylie Wagstaff Stephanie Corjon Nicholas Keiser Hiroshi Ochiai Zhongya Wang Julien Crettaz Annahita Keravala Sachiko Ohshima Piyanuch Wonganan Katye Fichter Eddy Kizana Alan Parker Lan Wu Daniel Gaddy Richard Koya Shondra Pruett Jiehua Zhou Bernhard Gentner Maria Limberis Garrett Rettig Karim Ghani Yemin Liu Efren Riu Jeffery Giering Angelo Lombardo Francesca Santoni de Sio The Excellence in Research Awards and Travel Grants were made possible through grant support from: 11

Outstanding New Investigator Award Winners The following four Outstanding New Investigator Award winners were selected by the Advisory Council based on merit and their contributions to the field of

13 Outstanding New Investigator Award Winners The following four Outstanding New Investigator Award winners were selected by the Advisory Council based on merit and their contributions to the field of gene therapy. The recipients will present their work at the special Outstanding New Investigator Symposium on Thursday, May 31 from 1:00 pm to 2:45 pm in Ballroom 6E. Akseli E. Hemminki, MD, PhD University of Helsinki Helsinki, Finland Oncolytic Adenoviruses for Killing of Cancer Initiating Cells Philip Ng, PhD Baylor College of Medicine Houston, Texas Gene Therapy with Helper-Dependant Adenoviral Vectors Derek Persons, MD, PhD St. Jude Children s Research Hospital Memphis, Tennessee Progress Toward Safe and Effective Gene Therapy for β-thalassemia and Sickle Cell Disease Gianpietro Dotti, MD Baylor College of Medicine Houston, Texas Genetic Modifications of T Cells for Adoptive Immunotherapy in Cancer Patients 12

14 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Faculty List as of May 1, 2007 Asa Abeliovich, MD, PhD Columbia University New York, NY Claudio Bordignon, MD Istituto Scientifico H. San Raffaele Milano, Italy Jay A. Chiorini, MD National Institutes of Health Bethesda, MD Gianpietro Dotti, MD Baylor College of Medicine Houston, TX Alan Aderem, PhD Institute for Systems Biology Seattle, WA Mirit I. Aladjem, PhD National Cancer Institute Bethesda, MD Steven M. Albelda, MD University of Pennsylvania Philadelphia, PA Andrea Amalfitano, DO, PhD Michigan State University East Lansing, MI Dale Ando, MD Sangamo BioSciences, Inc. Richmond, CA Tadashi Ariga, MD, PhD Hokkaido University Graduate School of Medicine Sapporo, Japan Andrew H. Baker, PhD BHF GCRC Glasgow, United Kingdom David Baltimore, PhD California Institute of Technology Pasadena, CA Krystof Bankiewicz, MD, PhD University of California San Francisco San Francisco, CA Michael A. Barry, PhD Mayo Clinic Rochester, MN Raymond T. Bartus, PhD Ceregene, Inc San Diego, CA Christopher Baum, MD Hannover Medical School Hannover, Germany Jean-Paul Behr, PhD Universite Louis Pasteur De Strasbourg Illkirch, France Ernest Beutler, MD The Scripps Institute La Jolla, CA Dawn E. Bowles, PhD Duke University Durham, NC Julie L. Boyer, PhD Weill Medical College of Cornell University New York, NY Xandra Breakefield, PhD Massachusetts General Hospital Charlestown, MA Malcolm K. Brenner, MD, PhD Baylor College of Medicine Houston, TX Dennis R. Burton, PhD Scripps Research Institute La Jolla, CA Andrew Byrnes, PhD Food & Drug Administration Rockville, MD Michele P. Calos, PhD Stanford University School of Medicine Stanford, CA Fabio Candotti, MD National Human Genome Research Institute Bethesda, MD Paula M. Cannon, PhD Childrens Hospital Los Angeles Los Angeles, CA Barrie J. Carter, PhD Targeted Genetics Corporation Seattle, WA Roberto Cattaneo, PhD Mayo Clinic Rochester, MN Marina Cavazzana-Calvo, MD, PhD Hopital Necker Enfants-Malades Paris, France Sunil Chada, PhD Introgen Therapeutics Inc. Houston, TX Jeffrey Chamberlain, PhD University of Washington School of Medicine Seattle, WA Chadwick Christine, MD University of California, San Francisco San Francisco, CA Mark J. Cooper, MD Copernicus Therapeutics, Inc. Cleveland, OH Kenneth Cornetta, MD Indiana University Indianapolis, IN Jacqueline Corrigan-Curay, MD, JD Office of Biotechnology Activities, NIH Bethesda, MD David Courtman, PhD St. Michael s Hospital Toronto, Canada Ronald G. Crystal, MD Weill Medical College of Cornell University New York, NY Beverly L. Davidson, PhD University of Iowa College of Medicine Iowa City, IA Mark M. Davis, PhD Stanford University Stanford, CA David A. Dean, PhD Northwestern University Medical School Chicago, IL Albert B. Deisseroth, MD, PhD Sidney Kimmel Cancer Center San Diego, CA David A. Dichek, MD University of Washington Seattle, WA Dennis Discher, PhD University of Pennsylvania Philadelphia, PA John Dong, MD, PhD Medical University of South Carolina Charleston, SC Ruxandra Draghia-Akli, MD, PhD ADViSYS, Inc Spring, TX Boro Dropulic, PhD Lentigen Corporation Baltimore, MD Dongsheng Duan, PhD University of Missouri School of Medicine Columbia, MO N. Matthew Ellinwood, PhD Iowa State University Ames, IA James Ellis, PhD Hospital for Sick Children Toronto, ON, Canada David W. Emery, PhD University of Washington Seattle, WA Howard J. Federoff, MD, PhD University of Rochester Rochester, NY Philip Felgner, PhD University of California, Irvine Irvine, CA Judah Folkman, MD Children s Hospital Boston Boston, MA Cornel Fraefel, PhD University of Zurich Zurich, Switzerland Svend O. Freytag, PhD Henry Ford Health System Detroit, MI Theodore Friedmann, MD University of California San Diego School of Medicine La Jolla, CA Yves Fromes, MD, PhD Institute of Myology-INSERM U582 Paris, France Louis P. Garrison, Jr., PhD University of Washington Seattle, WA 13

15 Faculty List Dan Gazit, DMD, PhD Hebrew University of Jerusalem Jerusalem, Israel Paloma H. Giangrande, PhD Duke University Durham, NC Nick Giannoukakis, PhD University of Pittsburgh Diabetes Institute Pittsburgh, PA Frank J. Giordano, MD Yale School of Medicine New Haven, CT Menzo Havenga, PhD Crucell Holland BV Leiden, Netherlands Robert G. Hawley, PhD The George Washington University Washington, DC Anthony Hayward, MD, PhD National Center for Research Resources Bethesda, MD Akseli E. Hemminki, MD, PhD University of Helsinki Helsinki, Finland Larry G. Johnson, MD University of Arkansas for Medical Sciences Little rock, AR Douglas J. Jolly, PhD Advantagene, Incorporated Encinitas, CA Karin Jooss, PhD Cell Genesys, Incorporated South San Francisco, CA Carl H. June, MD University of Pennsylvania Merion Station, PA Jeffrey H. Kordower, PhD Rush University Medical Center Chicago, IL Philip J. Laipis, PhD University of Florida Gainesville, FL Stephanie Laufs, PhD German Cancer Research Center Heidelberg, Germany Paola Leone, PhD University of Medicine and Dentistry of New Jersey Camden, NJ William Goins, PhD University of Pittsburgh School of Medicine Pittsburgh, PA Paola Grandi, PhD University of Pittsburgh School of Medicine Pittsburgh, PA John T. Gray, PhD St. Jude Childrens Research Hospital Memphis, TN Philip Greenberg, MD University of Washington Seattle, WA Markus Grompe, MD Oregon Health and Science University Portland, OR Dieter Gruenert, PhD California Pacific Medical Center Research Institute San Francisco, CA H. Kirk Hammond, MD University of San Diego and Veterans Affairs San Diego San Diego, CA Hideyoshi Harashima, PhD Hokkaido University Sapporo, Japan Kathryn Harris, PhD Office of Biotechnology Activities, NIH Bethesda, MD Mark Haskins, VMD, PhD University of Pennsylvania Philadelphia, PA Tina M. Henkin, PhD The Ohio State University Columbus, OH Terry Hermiston, PhD Berlex Biosciences San Pablo, CA Roland W. Herzog, PhD University of Florida Gainesville, FL Helen E. Heslop, MD Baylor College of Medicine Houston, TX Katherine A. High, MD The Children s Hospital of Philadelphia Howard Hughes Medical Institute Philadelphia, PA Daniel R. Hollyman, PhD Memorial Sloan-Kettering Cancer Center New York, NY Thomas J. Hope, PhD Northwestern University Feinberg School of Medicine Chicago, IL Leaf Huang, PhD University of North Carolina Chapel Hill School of Pharmacy Chapel Hill, NC Stephen Hyde, PhD University of Oxford Oxford, United Kingdom John Iacomini, PhD Brigham and Women s Hospital Boston, MA Michael Kaplitt, MD, PhD Weill Medical College of Cornell University New York, NY Noriyuki Kasahara, MD, PhD David Geffen School of Medicine at UCLA Los Angeles, CA Mark A. Kay, MD, PhD Stanford University School of Medicine Stanford, CA Cynthia Kenyon, PhD University of California San Francisco San Francisco, CA Hans-Peter Kiem, MD Fred Hutchinson Cancer Research Center Seattle, WA Nancy MP King, JD Wake Forest University School of Medicine Winston Salem, NC David H. Kirn, MD JENNEREX Biotherapeutics, Incorporated Montreal, Canada Jonathan Kimmelman, PhD McGill University San Francisco, CA Dwight Koeberl, MD, PhD Duke University Medical Center Durham, NC Donald B. Kohn, MD Childrens Hospital Los Angeles Los Angeles, CA Philip L. Leopold, PhD Weill Medical College of Cornell University New York, NY Bruce L. Levine, PhD University of Pennsylvania Philadelphia, PA Thomas K. Lewellen, PhD University of Washington Seattle, WA Shulin Li, PhD Louisiana State University Baton Rouge, LA Andre Lieber, MD, PhD University of Washington Seattle, WA Michael R. Linden, PhD Mount Sinai School of Medicine New York, NY Kristian H. Link, PhD Yale University New Haven, CT Dexi Liu, PhD University of Pittsburgh Pittsburgh, PA Pedro Lowenstein, MD, PhD Gene Therapeutics Research Institute Cedars Sinai Medical Center, University of California Los Angeles Los Angeles, CA Robert C. MacDonald, PhD Northwestern University Evanston, IL 14

16 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Faculty List Ian MacLachlan, PhD Protiva Biotherapeutics Incorporated Burnaby, Canada Harry L. Malech, MD National Institute of Allergy and Infectious Disease Laboratory of Host Defenses Bethesda, MD Punam Malik, MD Cincinnati Children s Hospital Cincinnati, OH Keith L. March, MD, PhD Indiana University Indianapolis, IN James Marks, MD, PhD University of California San Francisco San Francisco, CA Marcela Maus, MD, PhD Hospital of the University of Pennsylvania Wynnewood, PA Vivien Mautner, PhD CR UK Institute for Cancer Studies Birmingham, United Kingdom Fulvio Mavilio, PhD Dibit Inst Sci HS Raffaele Milan, Italy Anton P. McCaffrey, PhD University of Iowa Iowa City, IA Douglas M. McCarty, PhD Columbus Children s Research Institute Columbus, OH Paul B. McCray, Jr., MD University of Iowa College of Medicine Iowa City, IA Maritza C. McIntyre, PhD Biologics Consulting Group, Inc. Rockville, MD R. Scott McIvor, PhD University of Minnesota Minneapolis, MN John McPherson, PhD Genzyme Corporation Cambridge, MA Lali K. Medina-Kauwe, PhD Cedars-Sinai Medical Center Los Angeles, CA Carol H. Miao, PhD University of Washington Children s Hospital and Regional Medical Center Seattle, WA Hiroaki Mizukami, MD, PhD Jichi Medical School Minamikawachi, Tochigi, Japan Ryuichi Morishita, MD, PhD Osaka University Graduate School of Medicine Suita, Japan Bernard Moss, MD, PhD National Institute of Allergy and Infectious Disease Bethesda, MD Charles Murry, PhD University of Washington Seattle, WA Muna Naash, PhD University of Oklahoma Health Sciences Center Oklahoma City, OK Luigi Naldini, MD, PhD San Raffaele Telethon Institute for Gene Therapy (HSR - TIGET) Milano, Italy Philip Ng, PhD Baylor College of Medicine Houston, TX John J. Nemunaitis, MD Mary Crowley Medical Research Center Dallas, TX Eberhard Neumann, PhD Universitat Bielefeld Bielefeld, Germany Arthur W. Nienhuis, MD St. Jude Children s Research Hospital Memphis, TN Randolph J. Noelle, PhD Dartmouth College Hanover, NH Jan Nolta, PhD University of California at Shriners Hospital Sacramento, CA Masafumi Onodera, MD, PhD University of Tsukuba Tsukuba, Japan David Oupicky, PhD Wayne State University Detroit, MI Marco Passini, PhD Genzyme Corporation Framingham, MA Ralph W. Paul, PhD Targeted Genetics Corporation Seattle, WA Derek Persons, MD, PhD St. Jude Children s Research Hospital Memphis, TN Katherine Ponder, MD Washington University School of Medicine St. Louis, MO Matthew H. Porteus, MD, PhD University of Texas Southwestern Medical School Dallas, TX Jesus Prieto, MD, PhD Clinica Universitaria De Navarra Pamplona, Spain Suzie H. Pun, PhD University of Washington Seattle, WA Joseph Rabinowitz, PhD Thomas Jefferson University Philadelphia, PA Tony Reid, MD, PhD Moores University of San Diego Cancer Center San Diego, CA Kevin G. Rice, PhD University of Iowa College of Pharmacy Iowa City, IA Isabelle Riviere, PhD Memorial Sloan-Kettering Cancer Center New York, NY Ronald Rodriguez, MD, PhD Johns Hopkins Hospital Baltimore, MD Alain Rolland, PharmD, PhD Vical San Diego, CA Maria Grazia Roncarolo, MD, PhD San Raffaele Telethon Institute for Gene Therapy Milano, Italy Daniel Rosenblum, MD National Center for Research Resources Bethesda, MD John J. Rossi, PhD Beckman Research Institute City of Hope Duarte, CA Alexander Y. Rudensky, PhD University of Washington Seattle, WA Natalie Sacks, MD Cell Genesys, Incorporated South San Francisco, CA Michel Sadelain, MD, PhD Memorial Sloan-Kettering Cancer Center New York, NY R. Jude Samulski, PhD University of North Carolina at Chapel Hill Chapel Hill, NC Mark S. Sands, PhD Washington University School of Medicine St Louis, MO Bruno Sangro, MD, PhD University Clinic Pamplona, Spain Daniel Scherman, PhD INSERM U640 - CNRS UMR8151 Paris, France Allan Shipp Office of Biotechnology Activities, NIH bethesda, md Jonathan W. Simons, MD Winship Cancer Institute Atlanta, GA 15

17 Faculty List Vijay Singh, PhD Wave Biotech, LLC Somerset, NJ Evan Snyder, MD, PhD Burnham Institute for Medical Research La Jolla, CA George Stamatoyannopoulos, MD University of Washington Seattle, WA Duncan J. Stewart, MD, FRCPC St. Michael s Hospital Toronto, Canada Rainer F. Storb, MD Fred Hutchinson Cancer Research Center Seattle, WA David Strayer, MD, PhD Jefferson Medical College Philadelphia, PA Robert M. Strieter, MD, FCCP, FACP University of Virginia Health System Charlottesville, VA Barry Stripp, PhD University of Pittsburgh Pittsburgh, PA Erik S. G. Stroes, MD, PhD Amsterdam Molecular Therapeutics Amsterdam, Netherlands Bruce Sullenger, PhD Duke University Medical Center Durham, NC Francis C. Szoka, Jr., PhD University of California San Francisco San Francisco, CA Steve H. Thorne, PhD Stanford University Stanford, CA Adrian J. Thrasher, MD, PhD Institute of Child Health London, United Kingdom Alex W. Tong, PhD Baylor University Medical Center Dallas, TX Bruce E. Torbett, PhD, MSPH The Scripps Research Institute La Jolla, CA Christopher E. Touloukian, MD Indiana University School of Medicine Indianapolis, IN Tim Townes, PhD University of Alabama at Birmingham Birmingham, AL Cornelia L. Trimble, MD Johns Hopkins University Baltimore, MD David L. Urdal, PhD Dendreon Corporation Seattle, WA Victor W. van Beusechem, PhD VU University Medical Center Amsterdam, The Netherlands Richard G. Vile, PhD Mayo Clinic Rochester, MN Karen A. Vincent, PhD Genzyme Corporation Framingham, MA Samuel C. Wadsworth, PhD Genzyme Corporation Framingham, MA David J. Weber, MD, MPH University of North Carolina at Chapel Hill Chapel Hill, NC Lisa Wei, PhD GenVec Incorporated Gaithersburg, MD David B. Weiner, PhD University of Pennsylvania Philadelphia, PA Michael Welsh, MD University of Iowa College of Medicine Iowa City, IA Jeffrey A. Whitsett, MD Cincinnati Children s Hospital Medical Center Cincinnati, OH Carolyn Wilson, PhD U.S. Food and Drug Administration Center for Biologics Evaluation and Research Rockville, MD Christopher B. Wilson, MD University of Washington Seattle, WA William S. M. Wold, PhD St. Louis University School of Medicine St. Louis, MO John H. Wolfe, VMD, PhD Children s Hospital of Philadelphia Philadelphia, PA Jon A. Wolff, MD University of Wisconsin Madison Madison, WI Xiao Xiao, PhD University of North Carolina at Chapel Hill Chapel Hill, NC Masayuki Yamato, PhD Tokyo Women s Medical University Shinjuku-ku, Japan Feng Yao, PhD Brigham and Women s Hospital Boston, MA John A. Zaia, MD City of Hope National Medical Center Duarte, CA 16

18 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 General Meeting Information American Society of Gene Therapy Mission and Goals The American Society of Gene Therapy (ASGT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of gene and related cell and nucleic acid therapies and the promotion of professional and public education in the field. ASGT is the largest medical professional organization representing researchers and scientists dedicated to discovering new gene therapies. ASGT was established in 1996, and has grown to close to 2,500 members from around the world. The Society s goals are to: Promote Research, Development and Application Exchange Information and Promote Education Among Professionals and the Public Promote Development of Clinical Translations The ASGT 10th Annual Meeting will serve as a comprehensive forum to achieve these goals. Abstract Volume Molecular Therapy All abstracts accepted for presentation at the ASGT 10th Annual Meeting have been published in the May supplement of Molecular Therapy. Each attendee will received one copy of the supplement along with their registration materials. Admission Official name badges will be required for admission to all ASGT sessions. All Annual Meeting attendees receive a name badge with their registration bag. Name badges should be worn at all times inside the convention center, as badges will be used to control access to sessions and activities. Attendees are cautioned against wearing their name badges while away from the convention center as badges may draw unwanted attention to your status as visitors to Seattle. Admission by Ticket Only Some sessions at the Annual Meeting will require an additional ticket to control access and capacity. Tickets for these sessions are not included in the meeting s regular registration fees. Tickets will be collected at the door by ushers. There are still a limited number of tickets available at the Registration Desk. Meet-the-Investigator tickets are $20 and tickets to the 10th Anniversary Reception are $10. Business Center A full service FedEx Kinko s is available in the Washington State Convention & Trade Center. Open Monday at 7 a.m. thru Friday at 9 p.m. Open Saturday & Sunday 9 a.m. to 6 p.m. Telephone: (206) usa5161@fedexkinkos.com Web Site: Business Meeting Join the Society s leadership for a business meeting and continental breakfast. Saturday, June 2, :15 am 8:00 am (A light continental breakfast will be offered at 7:00 am) Ballroom 6ABC All members are welcome. See page 3 for more details. Camera/Recording Policy Due to privacy issues, it is the policy of ASGT that no cameras are permitted in the meeting sessions, exhibit hall, or poster sessions. Please refrain from taking any photos in those locations. Audio or videotaping is also prohibited. Cell Phones For the courtesy of all presenters and attendees, please ensure that cell phone ringers are turned off or silenced during all sessions. Committee Meetings Most standing and scientific committees will meet during the Annual Meeting. See page 3 for Committee Meeting information. Concierge & Visitor Center Seattle offers a wide variety of activities and restaurants to meet all tastes and budgets. The Citywide Concierge Center located at in the South Lobby, staffed by the Seattle Convention and Visitors Bureau, can help you with your city needs and reservations. Phone: (toll-free) or Concierge & Visitor Center Hours Wednesday, May 30 Thursday, June 1 Friday, June 2 Saturday, June 3 11:00 am 5:00 pm 7:00 am 5:00 pm 7:30 am 5:00 pm 7:30 am 5:00 pm 17

19 General Meeting Information continued Continuing Medical Education Accreditation/Credit Designation This activity has been planned and implemented in accordance with the Essential Areas and policies of the Accreditation Council for Continuing Medical Education through the joint sponsorship of the University of California, San Diego School of Medicine and the American Society of Gene Therapy. The University of California, San Diego School of Medicine is accredited by the ACCME to provide continuing medical education for physicians. The University of California, San Diego School of Medicine designates this educational activity for a maximum of AMA PRA Category 1 Credits TM. Physicians should only claim credit commensurate with the extent of their participation in the activity. NOTE: Sessions marked with an asterisk (*) are not part of the CME program. CME Certificates CME Certificates will be sent via mail after the meeting has concluded. All participants requiring CME credit must sign in at the CME desk on the day they are leaving the meeting to verify their attendance at the ASGT 10th Annual Meeting. The CME desk is located near the ASGT registration area in the South Lobby of the Washington State Convention & Trade Center. Cultural and Linguistic Competency Statement This activity is in compliance with California Assembly Bill 1195 which requires continuing medical education activities with patient care components to include curriculum in the subjects of cultural and linguistic competency. Cultural competency is defined as a set of integrated attitudes, knowledge, and skills that enables health care professionals or organizations to care effectively for patients from diverse cultures, groups, and communities. Linguistic competency is defined as the ability of a physician or surgeon to provide patients who do not speak English or who have limited ability to speak English, direct communication in the patient s primary language. Cultural and linguistic competency was incorporated into the planning of this activity. Additional resources on cultural and linguistic competency and information about AB1195 can be found on the UCSD CME website at Disclosure It is the policy of the University of California, San Diego School of Medicine to ensure balance, independence, objectivity, and scientific rigor. All faculty participating are required to disclose any real or apparent conflict of interest related to the content of their presentation. All faculty are also required to disclose any discussions of off label/ unapproved use of drugs or devices. Educational Methods and Materials Lectures, Case Presentations, Panel Discussions, Question and Answer Sessions, Audio/Video Presentations, Abstracts, Posters, Executive Summary. Educational Objectives At the conclusion of the activity, the participant should be able to: Discuss key aspects of the current laboratory and/or clinical research in gene therapy. Demonstrate knowledge of methods being used by gene therapy researchers and practitioners. Describe one or more stimulating new areas of inquiry in the area of gene therapy. Demonstrate familiarity with federal regulations and guidelines for clinical gene transfer studies. Evaluation Method Evaluation by questionnaire will address program content, presentation, and possible bias. Needs Clinical gene transfer has become more and more complex due to ongoing developments in the fields of gene therapy itself, together with bioethics, research integrity, and financial conflicts, as well as federal mandates, regulations and guidelines. Oligonucleotide Therapies, Novel Vector Development, Host-Vector Interactions and Vaccine Therapies will be discussed as well as many other scientific topics. This meeting will provide an educational forum for scientists and clinicians to expand their knowledge about the broad developments in these fields. Target Audience The target audience includes basic science and translational researchers, clinical investigators, physicians, postdoctoral fellows, graduate students, employees of federal government and regulatory agencies, and other healthcare professionals with an interest in the latest advancements in the field of gene therapy and related cell and nucleic acid therapies. Exhibits Exhibits will be located in Exhibit Halls 4AB, of the Washington State Convention & Trade Center. The companies, organizations and institutions exhibiting at the ASGT Annual Meeting will be providing you with the latest information on products and services directly related to your professional needs, as well as recruiting for open positions. An Exhibit Hall Social will be held on Wednesday evening and lunch will be offered on Friday in the exhibit hall. All participants are urged to allow adequate time in your daily schedule to visit the exhibits, as they are an integral part of the success of the meeting. 18

20 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 General Meeting Information continued Exhibit Hall Hours Exhibits will be open Wednesday, May 30 through Friday, June 1, Wednesday, May 30 Thursday, May 31 Friday, June 1 Friday, June 1 5:00 pm 9:00 pm 5:00 pm 8:00 pm 12:00 pm 2:00 pm 5:15 pm 8:15 pm Guest Attendance ASGT asks registered attendees to refrain from taking children, spouses, or guests to any educational session or functions offered at the 10th Annual Meeting. Internet Access & Policy Internet access will be available at the Washington State Convention & Trade Center to all meeting attendees. Please visit the internet kiosks located in Exhibit Hall 4AB. When others are waiting at the internet kiosks, please limit your Internet use to 15 minutes at a time so other attendees can access the internet as well. The WiFi lounge will allow those attendees with laptop computers wireless access in the Exhibit Hall 4AB. Please use the SSID of ASGT. Job Bank All openings posted as of May 11, 2007 on the ASGT Job Bank have been included in the Jobs Available Booklet found in the registration bags. Contact information is included in the booklet. Job postings for open positions and résumés may be posted on the Job Posting boards in Exhibit Hall 4AB. You are responsible for providing multiple copies of your posting. A number of tables and chairs will also be available in the job exchange center for interview use. Lead Retrieval A lead retrieval system has been made available to all exhibitors of the ASGT 10th Annual Meeting. Exhibitors may ask to scan attendee name badges with a hand held scanner in order to obtain attendee contact information. Materials Distribution Please refrain from distributing promotional materials throughout the meeting; these may only be distributed from an exhibit booth in Exhibit Hall 4AB. Media Members of the working media may register for the 10th Annual Meeting in the Press Room. Interview space, computers, telephones, and fax services are available for the convenience of media representatives covering the meeting. Press must register, provide credentials, and wear their press badge for admittance to ASGT sessions. Assistance will be provided to members of the media that would like to schedule interviews. No Smoking Smoking is prohibited at all 10th Annual Meeting sessions and events. Posters Abstract Posters will be on display in Hall 4AB. Thursday, May 31 Abstract Poster Session I 10:00 am 12:00 pm Poster Setup by Authors 12:00 pm 8:00 pm Poster Viewing (authors present from 5:00 pm 8:00 pm) 5:00 pm 8:00 pm Exhibit Hall Open 5:00 pm 8:00 pm Welcome Reception, Exhibits, and Poster Session 8:00 pm 8:30 pm Authors Remove Posters Friday, June 1 Abstract Poster Session II 7:00 am 12:00 pm Poster Setup by Authors 12:00 pm 8:15 pm Poster Viewing (authors present from 5:15 pm 8:15 pm) 12:00 pm 2:00 pm Exhibit Hall Open 12:15 pm 1:15 pm Lunch in Exhibit Hall 5:15 pm 8:15 pm Exhibit Hall Open 5:15 pm 8:15 pm Networking Reception, Exhibits, and Poster Session 8:15 pm 8:45 pm Authors Remove Posters Saturday, June 2 Abstract Poster Session III 7:00 am 12:00 pm Poster Setup by Authors 12:00 pm 7:00 pm Poster Viewing (authors present from 5:15 pm 7:00 pm) 5:15 pm 7:00 pm Poster Session 7:00 pm 7:30 pm Authors Remove Posters Press Room The Press Room is located in Washington State Convention & Trade Center, Level 6, Room 604. Press Room Hours Wednesday, May 30 Thursday, May 31 Friday, June 1 Saturday, June 2 3:00 pm 6:00 pm 7:45 am 5:00 pm 7:45 am 5:00 pm 7:45 am 5:00 pm 19

21 General Meeting Information continued Registration Desk Name badges, final programs, and abstract supplements will be distributed at the registration desk located in the South Lobby of the Washington State Convention & Trade Center. Registration Desk Hours Wednesday, May 30 Thursday, June 1 Friday, June 2 Saturday, June 3 Sunday, June 4 11:30 am 8:00 pm 7:00 am 5:00 pm 7:30 am 5:00 pm 7:30 am 5:00 pm 8:00 am 10:00 am Speaker Ready Room Hours Wednesday, May 30 Thursday, May 31 Friday, June 1 Saturday, June 2 Sunday, June 3 10:00 am 9:00 pm 6:30 am 7:00 pm 7:00 am 7:00 pm 7:00 am 6:00 pm 7:30 am 10:00 am Special Accessibility Needs If you require special accommodations in order to fully participate in the meeting, please visit the Registration Desk in the South Lobby. The ASGT staff will be happy to assist you with your specific needs. Shuttle Bus ASGT will not be providing shuttle transportation for the 10th Annual Meeting. The Washington State Convention & Trade Center is located within 6 blocks of the ASGT Annual Meeting hotel block. Speaker Ready Room The Speaker Ready Room is located in Washington State Convention & Trade Center, Level 6, Room 603. All faculty must deliver their presentations to the Speaker Ready Room the day before their session or at least four hours prior to their presentations. Equipment is available for faculty to review their materials. Audiovisual personnel will be available for assistance. Please mark your materials (your name, session and speaker order) so the materials can be returned to you. The Society strongly encourages faculty to pre-load presentation in the Speaker Ready Room; those faculty that load presentations in the meeting rooms during the sessions will have that time deducted from their presentation time by the Chair. ASGT strongly encourages faculty presenting on Thursday of the Annual Meeting to check-in at the Speaker Ready Room on Wed., May 30, to avoid congestion. Avoid delay and check-in early! 20

22 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Washington State Convention & Trade Center Floor Plans Level 1 Escalators up to asgt registration 21

23 Washington State Convention & Trade Center Floor Plans continued Level 2 LEVEL 2 asgt committee meetings meet the investigator sessions 22

24 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Washington State Convention & Trade Center Floor Plans continued Level 4 LEVEL 4 SKYBRIDGE ASGT REGISTRATION EXHIBITS INTERNET KIOSKS JOB EXCHANGE POSTERS SESSION ROOMS 10 TH ANNIVERSARY RECEPTION anniversary reception posters JOB EXCHANGE internet EXHIBITS REGISTRATION 23

25 Washington State Convention & Trade Center Floor Plans continued Level 6 plenary sessions level 6 ASGT session rooms (education, oral abstracts and scientific symposia) PLENARY sessions PRESS room room 604 speaker ready room room

26 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, Schedule-at-a-Glance (Room locations listed in detailed Program Schedule.) Wednesday, May 30 8:30 am 12:15 pm Board of Directors Meeting (Sheraton) 11:30 am 8:00 pm Registration Open 12:30 pm 3:30 pm Bioprocessing Symposium: Novel Technologies in the Manufacturing and Analysis of Gene Transfer Agents 12:30 pm 3:30 pm International Symposium: Gene Therapy Clinical Trials from Around the Globe 4:00 pm 5:30 pm Education Session I 4:00 pm 7:15 pm The NIH System of Oversight of Human Gene Transfer Research: Optimizing Science, Safety and Ethics 5:00 pm 9:00 pm Exhibit Hall Open 5:45 pm 7:15 pm Education Session II 7:00 pm 9:00 pm Exhibit Hall Social Thursday, May 31 7:00 am 5:00 pm Registration Open 8:00 am 9:15 am George Stamatoyannopoulos Lecture; Excellence in Research Awards Announced Keynote: Judah Folkman, MD, President, Children s Hospital Boston 9:30 am 11:30 am Scientific Symposia 10:00 am 12:00 pm Poster Setup by Authors 11:30 am 1:00 pm Lunch Break 11:45 am 1:00 pm Meet-the-Investigator Lunch Sessions 12:00 pm 8:00 pm Poster Viewing (authors present from 5:00 pm 8:00 pm) 1:00 pm 2:45 pm Outstanding New Investigator Symposium 3:00 pm 5:00 pm Oral Abstract Sessions 5:00 pm 6:00 pm Societies Presidents Meeting 5:00 pm 8:00 pm Exhibit Hall Open 5:00 pm 8:00 pm Welcome Reception, Exhibits, and Poster Session I 7:00 pm 10:00 pm Nonviral Vectors: 20 Year s Progress toward Gene Therapy Friday, June 1 7:00 am 12:00 pm Poster Setup by Authors 7:30 am 5:00 pm Registration Open 8:00 am 10:00 am Oral Abstract Sessions 10:15 am 12:15 pm Scientific Symposia 10:15 am 12:15 pm Clinical Trials Symposium 12:00 pm 2:00 pm Exhibit Hall Open 12:00 pm 8:15 pm Poster Viewing (authors present from 5:15 pm 8:15 pm) 12:15 pm 1:15 pm Lunch in the Exhibit Hall 1:15 pm 2:45 pm Oral Abstract Sessions 3:00 pm 5:15 pm Presidential Symposium Presidential Address: Theodore Friedmann, MD Keynote: Cynthia Kenyon, PhD, Professor, Biochemistry & Biophysics Department, University of California-San Francisco Top Four Abstract Presentations 5:15 pm 8:15 pm Exhibit Hall Open 5:15 pm 8:15 pm Networking Reception, Exhibits, and Poster Session II 7:00 pm 10:00 pm Corporate Symposium: DNA as a Drug Target: Engineered Zinc Finger Protein Therapeutics (Sangamo) Saturday, June 2 7:00 am 12:00 pm Poster Setup by Authors 7:15 am 8:00 am ASGT Business Meeting 7:30 am 5:00 pm Registration Open 8:00 am 10:00 am Scientific Symposia 10:15 am 12:15 pm Oral Abstract Sessions 12:00 pm 7:00 pm Poster Viewing (authors present from 5:15 pm 7:00 pm) 12:15 pm 2:00 pm ASGT Board of Directors Meeting 12:15 pm 2:00 pm Lunch Break 12:30 pm 1:45 pm Meet-the-Investigator Lunch Sessions 2:00 pm 5:00 pm Special 10th Anniversary Symposium Keynote Speakers: Ernest Beutler, MD, The Scripps Institute Maria Grazia Roncarolo, MD, PhD, San Raffaele Telethon Institute for Gene Therapy Malcolm K. Brenner, MD, PhD, Center for Cell and Gene Therapy, Baylor College of Medicine David Baltimore, PhD, President, California Institute of Technology 5:15 pm 7:00 pm Poster Session III 7:00 pm 9:00 pm 10th Anniversary Reception Sunday, June 3 8:00 am 10:00 am Registration Open 8:00 am 10:00 am Oral Abstract Sessions 10:00 am 10th Annual Meeting Concludes 25

27 Program Schedule WEDNESDAY, MAY 30 Wednesday, May 30 Early-Bird Session :30 pm - 3:30 pm Bioprocessing: Novel Technologies in the Manufacturing and Analysis of Gene Transfer Agents Room: 4C1/4C2 Chair Ralph W. Paul, PhD Speakers Vijay Singh, PhD Bioreactors for Gene and Cell Therapy Applications Alain Rolland, PharmD, PhD Analysis of Plasmid-Based Products: Development of a Novel Potency Assay Marcela Maus, MD, PhD Clinical Prospects for Genetically Engineered Artificial Antigen Presenting Cells Maritza C. McIntyre, PhD Process Development for Gene Therapy Products: Who, What, When, Where and How? Daniel R. Hollyman, PhD Scaled-Up Manufacturing of Genetically Modified T Lymphocytes for Phase I/II Clinical Trials Menzo Havenga, PhD The PER.C6 Cell Platform for Gene Therapy and Vaccine Products Early-Bird Session :30 pm - 3:30 pm International: Gene Therapy Clinical Trials from around the Globe Room: 4C3/4C4 Chair Jesus Prieto, MD, PhD Speakers Ryuichi Morishita, MD, PhD Favorable Results of Clinical Trials of HGF (Hepatocyte Growth Factor) Gene Therapy in Peripheral Artery Disease Vivien Mautner, PhD Suicide Gene Therapy using an Adenovirus Expressing Nitroreductase with the Prodrug CB1954: A Phase I/II Clinical Trial for Prostate Cancer Masafumi Onodera, MD, PhD Gene Therapy for Hematologic Malignancy Bruno Sangro, MD, PhD Gene Therapy of Liver Cancer using Adenoviral Vectors Tadashi Ariga, MD, PhD Hematopoietic Stem Cell (HSC) Gene Therapy for Two Patients with Adenosine Deaminase (ADA) without Myelopreparative Conditioning: A Suggestion for the Optimal Protocol for HSC Gene Therapy for ADA Deficiency Erik S.G. Stroes, MD, PhD Efficacy and Safety of Intramuscular Administration of AMT-010 (AAV-LPLS447X) in Lipoprotein Lipase Deficient Subjects 26

28 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Wednesday, May 30 Special Session 003 Organized by OBA, NIH 4:00 pm 7:15 pm The NIH System of Oversight of Human Gene Transfer Research: Optimizing Science, Safety and Ethics Room: 4C1/4C2 Segment I The Fundamentals of Oversight: NIH and Local Review Chair Allan Shipp Speakers Allan Shipp Welcome Remarks Allan Shipp Introduction to the NIH Office of Biotechnology Activities (OBA) and the Resources Offers Investigators Kathyrn Harris, PhD Overview of the Policy and Biosafety Framework for Human Gene Transfer Research: The NIH Guidelines for Research Involving Recombinant DNA Molecules Jacqueline Corrigan-Curay, MD, JD The What, When and How of Submitting Human Gene Transfer Protocols to the NIH OBA for Review by the Recombinant DNA Advisory Committee (RAC) Jacqueline Corrigan-Curay, MD, JD Safety and Annual Reporting; What to Report and How to Get the Most out of GeMCRIS, NIH OBA s Electronic Safety Reporting System Allan Shipp Institutional Biosafety Committees (IBCs): The Linchpin of Local Oversight Segment II Views from the Field David J. Weber, MD, MPH Biosafety Review of Human Gene Transfer: From Preclinical Studies to Human Trials John Zaia, MD An Investigator s Perspective: How to Prepare for RAC Review Nancy M.P. King, JD Informed Consent in Human Gene Transfer Trials Helen Heslop, MD, Nancy M.P. King, JD, Larry G. Johnson, MD The NIH RAC Review Process: Maximizing the Benefits All Speakers Ask the Faculty: A Final Opportunity to Ask Questions Education Sessions The goal of the ASGT Education Program is to provide participants with a broad background and introduction to topics relevant to the field of gene therapy. Education Sessions will be subdivided into two headings. Topical Review sessions will provide a broad perspective of an area of importance to the gene therapy community such as major vector systems, specific target organs or diseases, and funding/regulatory issues. Emerging Field Review sessions will provide a broad perspective of a technological or scientific discipline that has recently become of great significance to our members. Education Session 100 4:00 pm - 5:30 pm Emerging Field Review: Innate Immunity and Regulation of the Immune System Room: 606/607 Immune responses to vectors and transgene products are dependent on signals to the immune system in the target tissue at the time of gene transfer. The innate immune system has evolved to rapidly respond to such signals derived from the tissue or from pathogen-associated molecular patterns on viruses or nucleic acids. In the absence of such activation signals, antigen presentation may lead to activation of regulatory T cells rather than effector T cells, which can result in immune tolerance. This session will review activation signals, the innate immune system, and regulatory networks. Chair Roland W. Herzog, PhD Speakers Alan Aderem, PhD A Systems Approach to Dissecting Immunity Christopher B. Wilson, MD TLRs in Innate Immunity and the Induction of Adaptive Immunity Alexander Y. Rudensky, PhD Immunological Tolerance WEDNESDAY, MAY 30 27

29 Program Schedule continued WEDNESDAY, MAY 30 Wednesday, May 30 Education Session 101 4:00 pm - 5:30 pm Emerging Field Review: In Vivo Imaging Room: 608/609 In vivo imaging includes a group of technologies that are increasingly employed in pre-clinical studies and clinical trials. In vivo imaging techniques can provide information on vector distribution, transgene expression, and efficacy parameters such as metabolic activity or anatomic changes in animal models and patients. By employing non-invasive imaging techniques, experimental information can be obtained from individual patients or experimental animals over time and increase the range of outcome parameters that can be studied. This session will describe a range of techniques that are available for in vivo imaging in experimental and clinical settings. Chair Philip L. Leopold, PhD Speakers R. Scott McIvor, PhD Bioluminescence Imaging in Gene Transfer Studies Thomas K. Lewellen, PhD In Vivo Imaging Using PET and CT William Goins, PhD MRI Reporters for In Vivo Imaging Education Session 103 4:00 pm - 5:30 pm Topical Review: Retroviruses, Lentiviruses and Therapy for Hematologic Disease Room: Gene therapy has been most advanced for treatment of hematological diseases, including primary immune deficiencies, hemophilia, and hemoglobinopathies (sickle cell and thalassemia). Every job requires the right tool, and different vector strategies have been developed for each of these diseases. Three outstanding experts who are highly productive investigators in these areas will present the ABCs of the diseases and elucidate the gene therapy approaches that have been developed and evaluated to treat these conditions. Chair Donald B. Kohn, MD Speakers Fabio Candotti, MD Use of Retroviral/Lentiviral Vectors for the Treatment of Immune Deficiency Diseases Tim Townes, PhD Sickle Cell Disease and Thalassemia Katherine Ponder, MD Use of Retroviral and Lentiviral Vectors for the Treatment of Hemophilia Education Session 102 4:00 pm - 5:30 pm Topical Review: Non-viral Vector Design and Efficacy Room: The education session on nonviral gene therapy will feature three presentations emphasizing on fundamental aspects of nonviral gene therapy including plasmid-based and site-specific gene integration, intracellular processing and sub-cellular distribution of transgene, and clinical evaluation of lipoplexes by intravenous administration. Chair Dexi Liu, PhD Speakers Michele P. Calos, PhD Site-Specific Integration for Gene Therapy Hideyoshi Harashima, PhD Controlled Intracellular Trafficking of Non-viral Vector Ian MacLachlan, PhD Clinical Hurdles for Lipid Based Gene Transfer Education Session 104 4:00 pm - 5:30 pm Topical Review: Cancer Therapy Room: The Cancer Gene Therapy Education session will focus on three different gene-based cancer therapeutic approaches, vaccines, oncolytic viruses and the use of biological agents as systemic delivery vehicles of therapeutic agents. The speakers will provide the audience with a general overview for each approach, outlining the scientific rationale, discussing any historical experience and finishing with a brief glimpse of some current clinical trials where applicable. We invite all individuals interested in learning about the Cancer Gene Therapy arena to attend as these sessions should benefit both novices and experts alike! Chair Terry Hermiston, PhD Speakers: David H. Kirn, MD Targeted Oncolytic Viruses for Cancer: Clinical Trials Update Jonathan W. Simons, MD Tumor Vaccine Background and Results to Date with GVAX Studies Steve H. Thorne, PhD Use of Immune Cells as Carrier Vehicles for Delivery of Viral Cancer Therapies Break 5:30 pm 5:45 pm 28

30 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Wednesday, May 30 Education Session 110 5:45 pm - 7:15 pm Emerging Field Review: Strategies for Stem Cell Therapies Room: 606/607 In the last few years, transplantation of somatic stem cells, unmanipulated or genetically modified, has emerged as an entirely new medical technology with enormous therapeutic potential for neurodegenerative, cardiovascular, neuromuscular and metabolic disorders. Steady progress has been achieved in the identification of tissue-specific stem cells, in the study of their basic biological properties and function, and in the preclinical and early clinical analysis of their potential to repair, or replace, damaged tissues and organs in small and large animal models. This session will analyze the progress and prospects of stem cell transplantation in three important areas (central nervous system, heart and pancreas) with a specific focus on clinical application. Chair Fulvio Mavilio, PhD Speakers Evan Snyder, MD, PhD Understanding and Harnessing Stem Cell Biology: Development Programs and Cross-Talk Charles Murry, PhD Heart Regeneration by Stem Cell Transplantation Nick Giannoukakis, PhD Stem Cell Prospects for Diabetes Mellitus Education Session 112 5:45 pm - 7:15 pm Topical Review: AAV Vectors and Therapies for Monogenic Disorders Room: 608/609 Adeno-associated virus (AAV) has been on the central stage in gene therapy since ASGT s first annual meeting in Numerous progresses have been made in the last decade to move AAV gene transfer from cells in petri dish to cells in human body. In this education session, we will focus on novel methods to modify viral capsid and to expand AAV packaging capacity. Advances in these vector technologies have substantially expanded the territory of AAV gene therapy. The ultimate goal in AAV gene therapy is to treat human diseases. AAV has been the vector of choice for many monogenic disorders including cystic fibrosis, hemophilia and muscular dystrophy. In this education, we will use AAV-mediated muscular dystrophy gene therapy as an example to demonstrate the application of AAV vector in human diseases. Chair Dongsheng Duan, PhD Speakers Joseph Rabinowitz, PhD Methods to Generate and Application of Novel AAV Serotypes in Gene Therapy Jeffrey Chamberlain, PhD Gene Therapy Strategies for Muscular Dystrophy Using AAV Dongsheng Duan, PhD How to Design AAV Vectors to Overcome its Packaging Limitation WEDNESDAY, MAY 30 Education Session 111 5:45 pm - 7:15 pm Emerging Field Review: Regulatory RNAs Room: The knowledge base regarding the many roles of regulatory RNAs is rapidly increasing. Findings in this field have broad applications for understanding the regulation of gene expression. In addition, the field is producing important tools with therapeutic applications for the regulation of gene expression, studies of biologic function, and the delivery of nucleotide based therapeutics. The goal of this session is to give current overviews of the areas of RNA aptamers, riboswitches, micrornas, and RNAi. Chair Paul B. McCray, Jr., MD Speakers Paloma H. Giangrande, PhD RNA Aptamers - Basic Biology and Therapeutic Applications Tina M. Henkin, PhD Control of Gene Expression by RNA Structural Rearrangements: Riboswitches and RNA Sensors Paul B. McCray, Jr., MD micrornas and sirnas Education Session 113 5:45 pm - 7:15 pm Topical Review: Viral Vectors and Vaccines for Infectious Diseases Room: This session illustrates how understanding the biology of viruses guides engineering of replicating vectors for use as vaccines. Three classes of vectors will be covered: adenoviruses, negative strand RNA viruses, and vaccinia viruses. New vaccines against emerging diseases, multivalent vaccines, and the results of current animal and human trials based on replicating recombinant viruses will be discussed. Chair Roberto Cattaneo, PhD Speakers Michael A. Barry, PhD Adenoviral Vaccines Roberto Cattaneo, PhD Negative strand RNA Viruses Bernard Moss, MD, PhD Vaccinia Virus Vectors 29

Program Schedule continued THURSDAY, MAY 31 Thursday, May 31 Scientific Symposium 211 George Stamatoyannopoulos Lecture 200 8:00 am - 9:15 am George Stamatoyannopoulos Lecture Room: Ballroom 6ABC

Nienhuis, MD President-Elect Speaker Judah Folkman, MD Rationale for Anti-angiogenic Gene Therapy Presentation of Excellence in Research Awards Scientific Symposium 210 9:30 am - 11:30 am Cancer: Hot

31 Program Schedule continued THURSDAY, MAY 31 Thursday, May 31 Scientific Symposium 211 George Stamatoyannopoulos Lecture 200 8:00 am - 9:15 am George Stamatoyannopoulos Lecture Room: Ballroom 6ABC Chair Arthur W. Nienhuis, MD President-Elect Speaker Judah Folkman, MD Rationale for Anti-angiogenic Gene Therapy Presentation of Excellence in Research Awards Scientific Symposium 210 9:30 am - 11:30 am Cancer: Hot Topics in Cancer Gene Therapy Room: 4C1/4C2 Chair Steven M. Albelda, MD Speakers William S. M. Wold, PhD Role of the Immune System in Replicating Adenovirus using a Permissive Immune Competent Syrian Hamster Model Richard G. Vile, PhD Cell Carriers for the Systemic Delivery of Viral Vectors: The Best of Both Worlds Tony Reid, MD, PhD Analysis of Clinical Responses to Cancer Gene Therapy: Do the Current Standards Capture Clinically Relevant Responses Akseli E. Hemminki, MD, PhD Recent Progress in Development of Advanced Generation Oncolytic Adenoviruses 9:30 am - 11:30 am Clinical and Regulatory Affairs: Hot Topics in Late Stage Clinical Development and Regulation Room: 606/607 Chair Dale Ando, MD Speakers Natalie Sacks, MD Phase 3 Trials: GVAX Immunotherapy for Prostate Cancer Daniel Rosenblum, MD Resources for Gene Therapy Anthony Hayward, MD, PhD CTSA - A New Consortium to Advance Translational Research Carolyn Wilson, PhD An Update: FDA s FINAL Guidance for Industry: Gene Therapy Clinical Trials - Observing Participants for Delayed Adverse Events Scientific Symposium 212 9:30 am - 11:30 am Hemopoietic: Conditioning Regimens for Hemopoietic Gene Therapy Room: Ballroom 6E Chair Donald B. Kohn, MD Speakers Rainer F. Storb Nonmyeloablative Conditioning Regimens for Hematopoietic Cell Transplantation Hans-Peter Kiem, MD Hemopoietic Stem Cell Gene Transfer and Expansion Studies in Large Animal Models Claudio Bordignon, MD Stem Cell Gene Therapy Associated with Mild Conditioning for ADA SCID Harry L. Malech, MD Busulfan Conditioning Specifically Enhances Gene Therapy Making and Correction of Neutrophils and Monocytes in Chronic Granulomatous Disease 30

32 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Thursday, May 31 Scientific Symposium 213 9:30 am - 11:30 am Immunology of Gene Transfer: T Cell Biology Room: Chair John Iacomini, PhD Speakers Mark M. Davis, PhD T Cell Recognition by the Numbers Pedro Lowenstein, MD, PhD The Microanatomy of Anti-Viral Vector Immune Responses in the Brain: Therapeutic Implications John Iacomini, PhD T cell mediated Immunomodulation: Implications for Gene Therapy Roland W. Herzog, PhD Role of Regulatory T Cells in Immune Tolerance to the Transgene Product Scientific Symposium 214 9:30 am - 11:30 am Musculo-Skeletal: Innovations in Gene Therapy for Musculoskeletal Disorders Room: 608/609 Co-Chairs Jeffrey Chamberlain, PhD Dan Gazit, DMD, PhD Speakers Dongsheng Duan, PhD Gene Therapy for Dystrophin-Deficient Cardiomyopathy: Are We Ready? R. Jude Samulski, PhD AAV and Phase I DMD Studies Dwight Koeberl, MD, PhD Gene Delivery to Striated Muscle with AAV Vectors in Pompe Disease Dan Gazit, DMD, PhD Gene Transfer for Skeletal and Connective Tissue Repair: Innovations for In Vivo Detection and Imaging Scientific Symposium 215 9:30 am - 11:30 am Respiratory Tract: Hot Topics: Stem Cells, Cell-Based Therapies, and a Novel Animal Model of Cystic Fibrosis Room: Chair Stephen Hyde, PhD Speakers Barry Stripp, PhD Resident Stem Cells in Lung Disease: Repair and Potential for Therapeutic Interventions Robert M. Strieter, MD, FCCP, FACP Circulating Progenitor Cells Involved in Repair of the Lung in Response to Injury Michael Welsh, MD Developing a New Animal Model for Cystic Fibrosis Jeffrey Whitsett, MD Stem Cells and Regeneration of the Respiratory Epithelium Scientific Symposium 216 9:30 am - 11:30 am Viral: Mechanisms of Viral Vector Assembly and Processing Room: Chair Kenneth Cornetta, MD Speakers Cornel Fraefel, PhD Replication of HSV-1 and AAV Vectors Philip L. Leopold, PhD You Can t Judge a Cell by its Viral Receptors: Post-Entry Variations in Viral Vector Trafficking Paula M. Cannon, PhD Functional Requirements for HIV-1 and HIV-2 Assembly and Release Thomas J. Hope, PhD The Role of TRIM5a in Modulating Transduction of Cells with HIV- 2 Vectors Lunch Break 11:30 am 1:00 pm THURSDAY, MAY 31 31

33 Program Schedule continued THURSDAY, MAY 31 Thursday, May 31 Meet the Investigator :45 am - 1:00 pm Meet the Investigator Lunch Room: 213 Speakers Douglas J. Jolly, PhD Mark A. Kay, MD, PhD Career Development: Academic vs. Industry Meet the Investigator :45 am - 1:00 pm Meet the Investigator Lunch Room: 212 Speaker Beverly L. Davidson, PhD Getting Therapeutics to the Brain ASGT Outstanding New Investigator Symposium 230 1:00 pm 2:45 pm Special Session: Outstanding New Investigator Symposium Room: Ballroom 6E Co-Chairs Margaret A. Liu, MD Alan E. Smith, PhD Speakers Akseli E. Hemminki, MD, PhD Oncolytic Adenoviruses for Killing of Cancer Initiating Cells Philip Ng, PhD Gene Therapy with Helper-Dependant Adenoviral Vectors Derek Persons, MD, PhD Progress Toward Safe and Effective Gene Therapy for b-thalassemia and Sickle Cell Disease Gianpietro Dotti, MD Genetic Modifications of T Cells for Adoptive Immunotherapy in Cancer Patients Oral Abstract Session 240 3:00 PM 5:00 PM AAV Vector Integration and Immunology Room: 4C1/4C2 Co-Chairs Jay A. Chiorini, MD Douglas M. McCarty, PhD (Abstracts 1-8; see page 54) Oral Abstract Session 241 3:00 PM 5:00 PM Adenovirus Vectors: Immunity and Toxicology Room: Co-Chairs Andrew Byrnes, PhD Philip Ng, PhD (Abstracts 9-16; see page 54) Oral Abstract Session 242 3:00 PM 5:00 PM Physical Methods of Delivery Room: 4C3 Co-Chairs Ruxandra Draghia-Akli, MD, PhD Carol H. Miao, PhD (Abstracts 17-24; see page 55) Oral Abstract Session 243 3:00 PM 5:00 PM Neurologic I Room: Co-Chairs Krystof Bankiewicz, MD, PhD Michael Kaplitt, MD, PhD (Abstracts 25-32; see pages 55-56) Oral Abstract Session 244 3:00 PM 5:00 PM Cancer Immunotherapy: Dendritic Cells, Vaccines and Immune Modulation Room: 608/609 Co-Chairs Alex W. Tong, PhD Christopher E. Touloukian, MD (Abstracts 33-40; see page 56) Oral Abstract Session 245 3:00 PM 5:00 PM Cancer: Apoptosis & Suicide Room: 606/607 Co-Chairs Albert B. Deisseroth, MD, PhD Ronald Rodriguez, MD, PhD (Abstracts 41-48; see pages 56-57) 32

34 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Thursday, May 31 Oral Abstract Session 246 3:00 PM 5:00 PM Oncolytic Viruses Room: Co-Chairs Xandra Breakefield, PhD To be announced (Abstracts 49-56; see page 57) Oral Abstract Session 247 3:00 PM 5:00 PM Oligonucleotide Therapies I Room: 4C4 Co-Chairs Dieter Gruenert, PhD To be announced (Abstracts 57-64; see pages 57-58) Welcome Reception and Exhibits 5:00 pm 8:00 pm Room: Hall 4AB Poster Session I 5:00 pm 8:00 pm Room: Hall 4AB Integration and Genotoxicity (Abstracts 65-74; see page 58) AAV Production and Modification (Abstracts 75-92; see pages 58-59) Inborn Errors of Metabolism I Storage Diseases Plus Others (Abstracts ; see pages 59-60) Cardiovascular Gene Therapy I (Abstracts ; see pages 60-61) Gene Therapy for Muscular Dystrophies (Abstracts ; see pages 61-62) Vaccinating against Pathogens Using Gene Delivery (Abstracts ; see pages 62-63) Cancer Immunotherapy I (Abstracts ; see pages 63-65) Cancer: Apoptosis & Suicide (Abstracts ; see pages 65-67) Cancer: Targeted Therapy I (Abstracts ; see pages 67-68) Hematologic I (Abstracts ; see pages 68-69) Immune Responses to Transgene Products (Abstracts ; see pages 69-70) Gene Modification (Abstracts ; see pages 70-71) Stem Cell I (Abstracts ; see pages 71-72) Cell Processing Vector Production (Abstracts ; see pages 72-73) Special Session 250* 7:00 pm - 10:00 pm Nonviral Gene Transfer: 20 Year s Progress Toward Gene Therapy Room: Ballroom 6E Chair Francis C. Szoka, Jr., PhD Speakers Philip Felgner, PhD DOTMA Discovery and Its Impact on Nonviral Gene Delivery Jean-Paul Behr, PhD PEI Mediated Gene Transfer, What is Next? Eberhard Neumann Membrane Electroporation for Cancer Therapies and Direct Gene Electrotherapy Jon A. Wolff, MD Gene Transfer without a Carrier Leaf Huang, PhD Looking into the Future Friday, June 1 Oral Abstract Session 300 8:00 AM 10:00 AM RNA Vector Targeting, Expression and Integration Room: Co-Chairs Punam Malik, MD Bruce E. Torbett, PhD, MSPH (Abstracts ; see pages 73-74) Oral Abstract Session 301 8:00 AM 10:00 AM Adenovirus Vectors: Tropism, Receptors and Targeting Room: 4C1/4C2 Co-Chairs Michael A. Barry, PhD Lali K. Medina-Kauwe, PhD (Abstracts ; see page 74) FRIDAY, JUNE 1 33

35 Program Schedule continued FRIDAY, JUNE 1 Friday, June 1 Oral Abstract Session 302 8:00 AM 10:00 AM DNA Vectors for Non-Viral Gene Therapy Room: 608/609 Co-Chairs Michele P. Calos, PhD R. Scott McIvor, PhD (Abstracts ; see pages 74-75) Oral Abstract Session 303 8:00 AM 10:00 AM Inborn Errors of Metabolism I Room: 4C4 Co-Chairs Mark Haskins, VMD, PhD Mark S. Sands, PhD (Abstracts ; see page 75) Oral Abstract Session 304 8:00 AM 10:00 AM Cardiovascular Gene Therapy Room: 4C3 Co-Chairs Andrew H. Baker, PhD David A. Dichek, MD (Abstracts ; see pages 75-76) Oral Abstract Session 305 8:00 AM 10:00 AM Gene Transfer to Protect from Infectious Agents and Their Products Room: Co-Chairs Anton P. McCaffrey, PhD John A. Zaia, MD (Abstracts ; see page 76) Oral Abstract Session 306 8:00 AM 10:00 AM Cancer Immunotherapy: Receptor-Modified T Cells and T Cell Trafficking Room: 606/607 Co-Chairs Alex W. Tong, PhD Christopher E. Touloukian, MD (Abstracts ; see page 77) Oral Abstract Session 307 8:00 AM 10:00 AM Tumor Suppressor and Targeted Gene Therapy Room: Co-Chairs Noriyuki Kasahara, MD, PhD To be announced (Abstracts ; see pages 77-78) Scientific Symposium :15 am 12:15 pm Cardiovascular Gene Therapy: Gene and Cell Based Approaches to CHF Room: Chair Frank J. Giordano, MD Speakers H. Kirk Hammond, MD Adenylyl Cyclase Type VI Gene Transfer for CHF Frank J. Giordano, MD Therapeutic Targeting of Transcription to Treat and to Prevent Heart Failure Keith L. March, MD, PhD Adipose Derived Stem Cells Karen A. Vincent, PhD Angiogenesis Gene Therapy Using a Constitutively Active HIF-1alpha Construct for Peripheral Vascular and Coronary Artery Disease Clinical Trial Symposium :15 am - 12:15 pm Clinical Trial Symposium Room: Ballroom 6E Chair George Stamatoyannopoulos, MD Speakers Lisa Wei, PhD Clinical Trial in Age-related Macular Degeneration Raymond T. Bartus, PhD CERE -120 (AAV-Neurturin) for Parkinson s Disease: From Concept to Clinical Testing and Beyond Adrian J. Thrasher, MD, PhD Gene Therapy for SCID Bruce L. Levine, PhD Lentiviral Transduced Activated T Cell Infusions for HIV 34

36 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Friday, June 1 Clinical Trial Symposium :15 am - 12:15 pm Clinical Trial Symposium: Cancer Room: 4C1/4C2 Chair Helen E. Heslop, MD Speakers David H. Kirn, MD A Phase I-II Clinical Trial of JX-594, a GM-CSF-Armed Oncolytic Vaccinia Virus, in Patients with Advanced Liver Tumors John J. Nemunaitis, MD Use of a TGFβ2 Antisense Gene Modified Allogeneic Tumor Cell Vaccine in Non-Small Cell Lung Cancer Helen E. Heslop, MD Immunotherapy of Lymphoma using LMP2-Specific CTL s Svend O. Freytag, PhD Clinical Trials of Replication-Competent Adenovirus-Mediated Suicide Gene Therapy Scientific Symposium :15 am - 12:15 pm Embryonic/Somatic Stem Cell and Tissue Engineering Room: 606/607 Chair Michael R. Linden, PhD Speakers Dennis Discher, PhD Nature or Nurture at the Cellular Scale: Matrix Elasticity Directs Stem Cell Differentiation David Courtman, PhD Microvascular Regeneration in the Lung: A Novel Treatment for Established Pulmonary Hypertension Markus Grompe, MD Intrahepatic Stem Cells: Biology and Therapeutic Application Masayuki Yamato, PhD Cell Sheet Engineering for Regenerative Medicine Scientific Symposium :15 am - 12:15 pm Industrial Liaison: Drug Development and Gene Therapy Room: 608/609 Chair Douglas J. Jolly, PhD Speakers Douglas J. Jolly, PhD Integration of Gene Therapy and Drug Development David L. Urdal, PhD Development of Provenge (Sipuleucel-T), an Active Cellular Immunotherapy for Prostate Cancer Louis P. Garrison, PhD An Introduction to Pharmacoeconomics and Its Application in Drug Development and Gene Therapy John McPherson, PhD Autologous Chondrocyte Transplantation Carticel : Lessons Learned and Future Challenges Donald B. Kohn, MD ADA-Deficient SCID in the New World Scientific Symposium :15 am - 12:15 pm Non-Viral Gene Therapy: Toward Nonviral Gene Therapy Room: Chair Dexi Liu, PhD Speakers Robert C. MacDonald, PhD The Hydrophobic Portion of Lipofection Agents is Critical: Lessons from a Series of Cationic Phospholipid Derivatives Shulin Li, PhD Sequential Intramuscular Electro-Gene Therapy for Eliminating Systemic Tumors Mark J. Cooper, MD Nonviral Gene Transfer in the Lung: from Animals to Humans Dexi Liu, PhD Computer Controlled Hydrodynamic Injection for Localized Gene Delivery FRIDAY, JUNE 1 Exhibit Hall Open 12:00 pm 2:00 pm Lunch Break in the Exhibit Hall 12:15 pm 1:15 pm 35

37 Program Schedule continued FRIDAY, JUNE 1 Friday, June 1 Oral Abstract Session 320 1:15 PM 2:45 PM AAV Vector Biology Room: Co-Chairs Dawn E. Bowles, PhD Douglas M. McCarty, PhD (Abstracts ; see page 78) Oral Abstract Session 321 1:15 PM 2:45 PM HSV and Other DNA Viruses Room: 4C3 Co-Chairs Paola Grandi, PhD Feng Yao, PhD (Abstracts ; see page 78) Oral Abstract Session 322 1:15 PM 2:45 PM Non-Viral Delivery Strategies Room: 4C1/4C2 Co-Chairs Muna Naash, PhD Daniel Scherman, PhD (Abstracts ; see pages 78-79) Oral Abstract Session 323 1:15 PM 2:45 PM Inborn Errors of Metabolism II Room: 608/609 Co-Chairs N. Matthew Ellinwood, PhD Philip J. Laipis, PhD (Abstracts ; see page 79) Oral Abstract Session 325 1:15 PM 2:45 PM Lung and Respiratory Disease Room: Co-Chairs David A. Dean, PhD To be announced (Abstracts ; see page 80) Oral Abstract Session 326 1:15 PM 2:45 PM Stem Cells Translational Technology Session Room: Co-Chairs Markus Grompe, MD Jan Nolta, PhD (Abstracts ; see pages 80-81) Oral Abstract Session 327 1:15 PM 2:45 PM Cell Processing and Vector Production Room: 4C4 Co-Chairs Isabelle Riviere, PhD John T. Gray, PhD (Abstracts ; see page 81) Break 2:45 pm 3:00 pm Oral Abstract Session 324 1:15 PM 2:45 PM Gene Therapy for the Musculo Skeletal System Room: 606/607 Co-Chairs Yves Fromes, MD, PhD Jacques P. Tremblay, PhD (Abstracts ; see pages 79-80) 36

38 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Friday, June 1 Presidential Symposium 330 3:00 pm - 5:15 pm Presidential Symposium Room: Ballroom 6ABC Chair Theodore Friedmann, MD Speaker Cynthia Kenyon, PhD From Worms to Mammals: Genes that Control the Rate of Aging Presentation of Society Award Presentation of 4 Top Abstracts John Alexander - Cone-Targeted Gene Therapy by AAV Bernard Gentner, MD - MicroRNA-Regulated Lentiviral Vectors Drive Robust Tissue-, Lineage- and Developmental Stage-Specific Gene Expression in Hematopoietic, Neural and Embryonic Stem Cells Krys Bankiewicz, MD, PhD - CNS Viral Vector Delivery under Real-Time Imaging Control Michael C. Holmes, PhD - Targeted Disruption of CCR5 Using Engineered Zinc Finger Protein Nucleases Provides In Vivo Protection from HIV Networking Reception, Exhibits 5:15 pm 8:15 pm Room: Hall 4AB Poster Session II 5:15 pm 8:15 pm Room: Hall 4AB Adenovirus Vectors: Applications (Abstracts ; see pages 81-82) HSV and Other DNA Viruses (Abstracts ; see page 83) DNA Vectors for Non-Viral Gene Therapy (Abstracts ; see page 84) Physical Methods of Delivery I (Abstracts ; see pages 84-85) Non-Viral Vectors: Intracellular Trafficking (Abstracts ; and Abstract 741 see pages 85-86) Neurologic I (Abstracts ; see pages 86-88) Gene Therapy of Bone and Cartilage (Abstracts see page 88) Protecting against Infectious Agents Using Gene Transfer (Abstracts ; see pages 88-90) Cancer Immunotherapy II (Abstracts ; see pages 90-91) Cancer: Targeted Therapy II (Abstracts ; see pages 91-93) Cancer: Targeted Therapy III (Abstracts ; see pages 93-94) Hematologic II (Abstracts ; see pages 94-95) Immune Responses to Adenoviral Vectors (Abstracts ; see page 95) Gene Expression in Animal Model Systems (Abstracts ; see pages 95-97) Oligonucleotide Therapies I (Abstracts ; see pages 97-98) FRIDAY, JUNE 1 37

39 Program Schedule continued Friday, June 1 Saturday, June 2 FRIDAY, JUNE 1 Corporate Symposium 340* Organized by Sangamo BioSciences, Inc. 7:00 pm 10:00 pm DNA as a Drug Target: Engineered Zinc Finger Protein Therapeutics Room: Ballroom 6E Program: Zinc finger DNA binding proteins (ZFPs) can be engineered to create transcription factors that up- or down- regulate the expression of endogenous genes (ZFP TFs) and targeted nucleases (ZFNs) designed to make specific cuts in the genome to facilitate gene disruption, gene correction and targeted gene addition. These functions all have potential therapeutic applications. A Phase 2 human clinical trial is in progress to test a ZFP TF activator of VEGF for the treatment of diabetic neuropathy and Phase 1 studies have been initiated to test this ZFP TF for therapeutic angiogenesis. The goal of this program is to provide an overview of ZFPs, ZFP TFs and ZFNs and the clinical and preclinical data that support their potential applications as novel therapeutics that act at the level of the cell s DNA. Chair: Donald Kohn, MD The Saban Research Institute of Children s Hospital Los Angeles Speakers: ZFPs as Therapeutics - Applications and Advantages of the Technology Philip Gregory, PhD Sangamo BioSciences, Inc. Therapeutic Applications of a ZFP TF activator of VEGF-A Dale Ando, MD Sangamo BioSciences, Inc. ZFN-mediated Therapeutic Gene Disruption and Targeted Gene Addition Philip Gregory, PhD Sangamo BioSciences, Inc. Michael Jensen, MD City of Hope, Beckman Research Institute Carl June, MD Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine Luigi Naldini, MD, PhD The San Raffaele Telethon Institute for Gene Therapy (HSR- TIGET) ASGT Business Meeting Room: Ballroom 6ABC 7:15 am 8:00 am Scientific Symposium 400 8:00 am - 10:00 am Ethics: Round Table Discussion on the Ethics of Patient Selection Room: 608/609 Chair Punam Malik, MD Speakers Barrie J. Carter, PhD Industry Perspective on Recruitment of Subjects for Clinical Trials Jonathan Kimmelman, PhD Justice and Subject Selection in Gene Transfer Trials Marina Cavazzana-Calvo, MD, PhD Ethics of Patient Selection for Severe Immunodeficiencies Additional Round Table Participants Margaret Rosenfeld, MD, MPH, Mark Guntrum Scientific Symposium 401 8:00 am - 10:00 am Gene Therapy for Genetic Diseases: Immune Responses to Gene Therapy of Genetic Diseases Room: Chair Andrea Amalfitano, DO, PhD Speakers Katherine A. High, MD Human Responses to AAV Capsid: Lessons from Clinical Studies Katherine Ponder, MD Immune Responses after Gene Therapy for Mucopolysaccharidosis Randolph J. Noelle, PhD Cellular Networks in Immune Suppression Luigi Naldini, MD, PhD Innate and Adaptative Immune Response to Lentiviral Vectors: How to Evade Them and Improve the Efficacy of Gene Therapy 38

40 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Saturday, June 2 Scientific Symposium 402 8:00 am - 10:00 am Gene-Based Vaccines: Engineered Lymphocytes for Cancer and HIV Room: Ballroom 6E Chair Carl H. June, MD Speakers Philip Greenberg, MD Engineering T Cells with a Second TCR: Imparting Specificity and Improving Function Michel Sadelain, MD, PhD Targeting Tumors with Genetically Enhanced T Lymphocytes Carl H. June, MD Engineered T Cells for Therapy of Cancer Cornelia L. Trimble, MD HPV Vaccines: The Beginning of the End of Cervical Cancer Scientific Symposium 404 8:00 am - 10:00 am Neural Disorders: Parkinson s Disease: From Genetics to the Clinic Room: Chair John H. Wolfe, VMD, PhD Speakers Asa Abeliovich, MD, PhD Molecular Mechanisms of Parkinson s Disease Howard J. Federoff, MD, PhD Therapeutic Insights Learned from Parkinson s Disease Genetics Raymond T. Bartus, PhD (Replaces J. Kordower) Gene Therapy for Parkinson s Disease: Preclinical Studies and Clinical Considerations Chadwick Christine, MD Gene Therapy for Parkinson s Disease: Tuning Basal Ganglia Function Enzymatically? Scientific Symposium 403 8:00 am 10:00 am Infectious Disease: Rapid Response to Infection: Genetic Approaches to Passive Protection Room: 606/607 Co-Chairs Julie L. Boyer, PhD Boro Dropulic, PhD Speakers Ronald G. Crystal, MD Gene Transfer Directed Antibody Therapy Karin Jooss, PhD Stable, High-Level Expression of Full-Length Antibodies using the 2A Peptide Dennis R. Burton, PhD Human Antibodies as Anti-Virals James Marks, MD, PhD Use of Molecular Evolution for Developing Antibodies to Botulinum Neurotoxin Scientific Symposium 405 8:00 am - 10:00 am Oligonucleotide Based Therapies: Diversity of Oligonucleotides in Gene Therapy Room: Ballroom 6A Chair Matthew H. Porteus, MD, PhD Speakers Paloma H. Giangrande, PhD Aptamers Come of Age: New Applications of RNA Aptamers Kristian H. Link, PhD Engineering High Speed Allosteric Ribozymes for Gene Control John J. Rossi, PhD RNA Oligomer Based Gene Therapy for the Treatment of HIV Infection Bruce Sullenger, PhD Anti-Blood Clotting Aptamers Break 10:00 am 10:15 am SATURDAY, JUNE 2 39

41 Program Schedule continued SATURDAY, JUNE 2 Saturday, June 2 Oral Abstract Session :15 AM 12:15 PM AAV Vector Application and Tropism Room: Co-Chairs Dongshen Duan, PhD Hiroaki Mizukami, MD, PhD (Abstracts ; see pages 98-99) Oral Abstract Session :15 AM 12:15 PM Adenovirus Vectors: Cancer Therapy Room: 608/609 Co-Chairs Andre Lieber, MD, PhD Victor W. van Beusechem, PhD (Abstracts ; see page 99) Oral Abstract Session :15 AM 12:15 PM Intracellular Trafficking of Non-Viral Vehicles Room: 4C4 Co-Chairs David Oupicky, PhD Theresa M. Reineke, PhD (Abstracts ; see pages ) Oral Abstract Session :15 AM 12:15 PM Neurologic II Room: Co-Chairs Marco Passini, PhD To be announced (Abstracts ; see page 100) Oral Abstract Session :15 AM 12:15 PM Vaccination Via Gene Delivery Room: 4C-3 Co-Chairs David Strayer, MD, PhD David B. Weiner, PhD (Abstracts ; see pages ) Oral Abstract Session :15 AM 12:15 PM Cancer Gene Therapy Room: 606/607 Co-Chairs Sunil Chada, PhD John Dong, MD, PhD (Abstracts ; see pages ) Oral Abstract Session :15 AM 12:15 PM Hematopoietic Stem Cell Gene Therapy Room: 4C1/4C2 Co-Chairs Robert G. Hawley, PhD Derek Persons, MD, PhD (Abstracts ; see page 102) Oral Abstract Session :15 AM 12:15 PM Gene Modification Room: Co-Chairs Christopher Baum, MD Adrian J. Thrasher, MD, PhD (Abstracts ; see pages ) Lunch Break 12:15 pm 2:00 pm Meet the Investigator :30 pm - 1:45 pm Meet the Investigator Lunch Room: 213 Speakers Jan Nolta, PhD Samuel C. Wadsworth, PhD Career Development: Academic vs. Industry Meet the Investigator :30 pm - 1:45 pm Meet the Investigator Lunch Room: 212 Speaker Markus Grompe, MD Gene Therapy for Liver Disease 40

Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Saturday, June 2 10th Anniversary Symposium 430 2:00 pm - 5:00 pm 10th Anniversary Symposium: A

Problem Maria Grazia Roncarolo, MD, PhD Gene Therapy for Immuno Deficiency Diseases: From Basic Discoveries to Cure Malcolm K.

Vectors: Novel Vectors and Applications (Abstracts 782-803; see pages 103-104) RNA Vector Production and Targeting (Abstracts 804-818; see pages 104-105) AAV Application I (Abstracts 819-838; see

(Abstracts 877-892; see pages 108-109) Non-Viral Delivery Strategies (Abstracts 893-908; see pages 109-110) Physical Methods of Delivery II (Abstracts 909-924; see pages 110-111) Non-Viral Vectors:

(Abstracts 969-983; see page 114) Neurologic II (Abstracts 984-1003; see pages 115-116) Lung and Respiratory Disease (Abstracts 1004-1018; see pages 116-117) Oligonucleotide Therapies II (Abstracts

42 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Program Schedule continued Saturday, June 2 10th Anniversary Symposium 430 2:00 pm - 5:00 pm 10th Anniversary Symposium: A Decade from Promise to Clinical Reality Room: Ballroom ABC Chair Theodore Friedmann, MD Speakers Ernest Beutler, MD Phenotypic Variability in Single Gene Diseases: Seeking a Solution for an Unsolved Problem Maria Grazia Roncarolo, MD, PhD Gene Therapy for Immuno Deficiency Diseases: From Basic Discoveries to Cure Malcolm K. Brenner, MD, PhD Implementing Gene Therapies for Complex Disorders David Baltimore, PhD Gene Therapy for Cancer and Infectious Diseases Poster Session III 5:15 pm 7:00 pm Room: Hall 4AB RNA Virus Vectors: Novel Vectors and Applications (Abstracts ; see pages ) RNA Vector Production and Targeting (Abstracts ; see pages ) AAV Application I (Abstracts ; see pages ) AAV Application II (Abstracts ; see pages ) Adenovirus Vectors: Targeting and Host Response (Abstracts ; see pages ) Adenovirus Vectors: New Technology (Abstracts ; see pages ) Non-Viral Delivery Strategies (Abstracts ; see pages ) Physical Methods of Delivery II (Abstracts ; see pages ) Non-Viral Vectors: Biomaterials and Formulations (Abstracts ; see pages ) Inborn Errors of Metabolism II Hemophilia Plus Others (Abstracts ; see pages ) Cardiovascular Gene Therapy II (Abstracts ; see page 114) Neurologic II (Abstracts ; see pages ) Lung and Respiratory Disease (Abstracts ; see pages ) Oligonucleotide Therapies II (Abstracts ; see page 117) Stem Cell II (Abstracts ; see page 118) 7:00 pm 9:00 pm Special 10 th Anniversary Reception Skybridge/Room 4D Tickets required (available at ASGT Registration Desk) Join us for a special reception to help celebrate the 10 th Anniversary Meeting of the American Society of Gene Therapy. SATURDAY, JUNE 2 10th Anniversary Symposium & Reception are supported by 41

43 Program Schedule continued Sunday, June 3 Oral Abstract Session 500 8:00 AM 10:00 AM Clinical Gene Therapy Oral Abstract Session Room: 4C1/4C2 Co-Chairs Mark A. Kay, MD, PhD Katherine Ponder, MD (Abstracts ; see page 119) Oral Abstract Session 501 8:00 AM 10:00 AM RNA Viral Vectors: Genotoxicity and Integration Room: Co-Chairs David W. Emery, PhD Stephanie Laufs, PhD (Abstracts ; see page 119) Oral Abstract Session 502 8:00 AM 10:00 AM Synthetic Vehicles for In Vivo Applications Room: 4C-4 Co-Chairs Kevin G. Rice, PhD Suzie H. Pun, PhD (Abstracts ; see page 120) Oral Abstract Session 503 8:00 AM 10:00 AM Gene and Cell Therapies for Muscular Dystrophies Room: 4C-3 Co-Chairs Paul D. Robbins, PhD Xiao Xiao, PhD (Abstracts ; see page 120) Oral Abstract Session 504 8:00 AM 10:00 AM Vector and Transgene Immunology Room: 606/607 Co-Chairs Paola Leone, PhD R. Jude Samulski, PhD (Abstracts ; see page 121) Oral Abstract Session 505 8:00 AM 10:00 AM Gene Expression in Animal Model Systems Room: Co-Chairs Mirit I. Aladjem, PhD James Ellis, PhD (Abstracts ; see page 121) Oral Abstract Session 506 8:00 AM 10:00 AM Oligonucleotide Therapies II Room: Co-Chairs Anton P. McCaffrey, PhD Bruce Sullenger, PhD (Abstracts ; see page 122) Oral Abstract Session 507 8:00 AM 10:00 AM Stem Cells Basic Science Session Room: 608/609 Co-Chairs Gerhard Bauer, PhD Barbara Engel, MD, PhD (Abstracts ; see page 122) 10th Annual Meeting Concludes 10:00 am SUNDAY, JUNE 3 42

44 ASGT 2007 Annual Meeting Washington State Convention & Trade Center Seattle, WA, USA Exhibit Hall Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3,

45 Exhibitor Descriptions Aldevron, LLC th Street South Fargo, ND Telephone: Fax: Website: Booth Number: 208 Aldevron manufactures plasmid-dna for diagnostic, preclinical and clinical applications. Our GMP-source service is designed to produce DNA for clinical-grade virus (e.g. AAV) production. Aldevron s GIA service allows clients to test advanced gene-delivery technologies including electroporation and to generate antibodies to any antigen. Finally, over 40 expression, cytokine and reporter plasmids are available at amaxa GmbH Nattermannallee 1 Koeln, NRW Germany Telephone: +49(0) Fax: +49(0) scientific-support@amaxa.com Website: Booth Number: 124 amaxa develops and commercializes innovative products for the transfer of DNA or sirna into cells. Its unique Nucleofector technology provides a breakthrough tool for the highly efficient, nonviral transfection of primary cells and hard-to-transfect cell lines. The recently introduced Nucleofector 96-well Shuttle System now also allows high throughput transfection in 96-well format. American Society of Gene Therapy 555 East Wells Street, Suite 1100 Milwaukee, WI Telephone: Fax: info@asgt.org Website: Booth Number Stop by the ASGT membership booth to pick-up membership pin, Abstracts on Disc, membership applications and other Society information. Available at the booth for purchase are fashionable ASGT logo polo shirts! Amnis Corporation 2505 Third Ave, Suite 210 Seattle, WA Telephone: Fax: gpage@amnis.com Website: Booth Number: 304 Amnis Corporation offers the ImageStream imaging flow cytometry system, which combinesthe power of quantitative image analysis and flow cytometry in a single platform. Measure the amount, location, distribution and movement of your target molecules on, in or between cells. Generate data from tens of thousands of cells to insure statistical accuracy. Bentham Science Publishers, Ltd. R-103 Zoramin Residency, Scheme 33 Gulzar-e-Hejri, University Road Karachi, Sindh Pakistan Telephone: Fax: marketing@benthamscience.org Website: Booth Number: 312 A major STM journal publisher of 79 titles in 2007, answers the information needs of the pharmaceutical, bio and medical research community. Leading journals include Current Drug Metabolism (Impact Factor 5.4) and Current Pharmaceutical Design (Impact Factor 4.8): FREE online journals and information: Subscribe, Consortia, discounted global licenses and trials: subscriptions@bentham.org BioLife Solutions, Inc North Creek Parkway North Bothell, WA Telephone: Fax: msnyder@biolifesolutions.com Website: Booth Number: 301 BioLife, a leader in biopreservation technologies, provides fullydefined, serum-free, protein-free preservation media. CryoStor and HypoThermosol solutions represent the next generation of Cryopreservation and Hypothermic storage media. These cgmp solutions provide: Improved Post-storage survival, more Rapid Recovery of cells/tissues, Higher Yields, Faster Cell Attachment and Reduced postthaw cell death (apoptosis/necrosis). 44

46 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Exhibitor Descriptions BioSpherix, LTD 6678 Route 17 Redfield, NY Telephone: Fax: bbixby@biospherix.com Website: Booth Number: 223 X-VIVO is the first closed-incubation-system. Consists of multiple independent, high-performance, O2/CO2 incubators that open only into aseptic isolation cabinets with identical atmospheres for uninterrupted conditions during cell handling. The BioSpherix X-VIVO can be a cgmp compliant mini-facility, offering cell therapists an economical and practical alternative to expensive walk-in clean rooms. Boehringer Ingelheim Binger Strasse 173 Ingelheim, Rhineland Palatinate Germany Telephone: biopharma@bc.boehringer-ingelheim.com Website: Booth Number: The Boehringer Ingelheim Corporation is one of the world s 20 leading pharmaceutical companies. We offer the entire technology chain for development and production of biopharmaceuticals like antibodies and fragments thereof, proteins, scaffolds and pdna, in a one-stop shop at our facilities in Austria (microorganisms) and Germany (mammalian cell culture) with additional fill&finish capacity in the U.S. BTX/Harvard Apparatus 84 October Hill Road Holliston, MA Telephone: Fax: techsupport.btx@harvardapparatus.com Website: Booth Number: 104 BTX offers products for Electroporation & Electrofusion applications for in vitro, in vivo and High Throughput. We are introducing two new product lines to our family of BTX products. The Micorporator can transfect difficult cells in small volumes and the ECIS for monitoring cell growth, movement, morphology. CellGenix, GmbH 602 Hillside Ave. Antioch, IL Telephone: Fax: cellgenixusa@aol.com Website: cellgenix.com Booth Number: 316 CellGenix manufactures high quality cgmp and research grade cytokines and culture medium for dendritic, stem, and T cell manipulations. We also offer a line of GMP culture bags made from clear, inert, gas permeable FEP plastic and provide tailor-made cell and protein therapeutics for cancer treatment and orthopedic surgery. Center for Cellular and Molecular Therapeutics, The Children s Hospital of Philadelphia 302 Abramson Pediatric Research Center 3516 Civic Center Blvd. Philadelphia, PA Telephone: Fax: sunj@ .chop.edu Website: Booth Number: 322 The Center for Cellular and Molecular Therapeutics is dedicated to the understanding, development and application of gene and related cell and nucleic acid therapies. Since its inception, the Center has developed a cgmp vector production facility. The facility has successfully formulated clinical graded vectors, and was recently awarded an NHLBI contract for manufacturing clinical graded AAV vectors. Cogenics 100 Perimeter Park, Suite C Morrisville, NC Telephone: Fax: info@cogenics.com Website: Booth Number: 224 Cogenics, A division of Clinical Data, provides a comprehensive range of molecular and pharmacogenomics services for both research and regulated environments, including nucleic acid extraction, genotyping, sequencing, gene expression, and biorepository. 45

47 Exhibitor Descriptions Corning Incorporated One Riverfront Plaza MP-HQ-E1 Q21A Corning, NY Telephone: Fax: Website: Booth Number: 302 A diversified technology company; manufactures high quality, high performance research products for life sciences. Corning will exhibit reliable and fully-tested cell culture products for scale up. Included will be CellSTACK Culture Chambers, E-Cube Culture System, Baffled Erlenmeyer Flasks and Disposable Spinner Flasks. Key products from first culture to high throughput screening will also be on display. Dataworks Development, Inc. P.O. Box 174 Mountlake Terrace, WA Telephone: Fax: rick@dwdev.com Website: Booth Number: 219 Freezerworks 5 and Freezerworks Unlimited freezer inventory software tracks samples across multiple freezers while managing workflow. Safeguard data with comprehensive security features, 21 CFR part 11 compliance, and cryogenic-safe bar code labeling. Import and web service features assist integration with current laboratory processes. For all freezers and tanks. DNAVEC Corporation Kannon-dai Tsukuba, Ibaraki Japan Telephone: Fax: info@dnavec-corp.com Website: Booth Number: 116 DNAVEC Corporation developed novel RNA virus vector (Sendai virus vector) for gene therapy and genetic vaccines and unique pseudotyped lentivirus vector for use in respiratory system. We would like to find collaborative researchers who are interested in using our vector technology for basic studies and/or clinical application. EUFETS AG Vollmersbachstr. 66 Idar-Oberstein Germany Telephone: Fax: eufets@fresenius-biotech.com Website: Booth Number: 120 UFETS AG (Germany) is a contract manufactoring organisation focussing on the development and cgmp production for cell and gene therapy, especially viral vectors and gene modified cells. EUFETS supports product development (R&D / GLP studies), clinical trials (phase I to III) as well as small market supply. FightSMA 1807 Libbie Avenue, Suite 104 Richmond, VA Telephone: Fax: heatherlennon@fightsma.com Website: Booth Number: 324 FightSMA is a non-profit organization dedicated to accelerating a cure and treatment for Spinal Muscular Atrophy (SMA) which kills more babies than any other genetic disease. FightSMA has raised more than $6 million since it was founded and has funded research at more than 40 institutions, both domestic and international. Geneart Inc. 28 Kirk Bradden Road East M8Y 2E6 Etobicoke Toronto, ON M842E6 CANADA Telephone: Fax: Website: Booth Number: 323 Based on a gene synthesis technology platform, GENEART provides integrated system solutions for DNA engineering and processing to deliver improved drugs and biotech products to academia and pharmaceutical industries. The service portfolio ranges from gene optimization over synthetic gene production, gene variants and gene libraries to highest quality plasmid production and DNA-based agents. 46

48 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Exhibitor Descriptions Greater Boston Convention & Visitors Bureau Two Copley Place, Suite 105 Boston, MA Phone: Fax: tbodden@bostonusa.com Website: Booth Number: 419 The Greater Boston Convention & Visitors Bureau is a not for profit organization that promotes and markets the Greater Boston area to visitors and convention attendees. We offer an array of services that include information on hotels, restaurants, retail outlets, tours and local attractions. For more information, please visit the web site www. bostonusa.com. Ichor Medical Systems, Inc Nancy Ridge Drive, Suite 107 San Diego, CA Telephone: x108 Fax: mdelasalas@ichorms.com Website: Booth Number: 112 Ichor has developed a powerful, clinical electroporation system for nonviral DNA delivery for applications that include therapeutic cancer vaccines, therapeutic proteins and vaccines for infectious diseases. Ichor s TriGrid(tm) Delivery System integrates several patented technologies into a simple handheld device enabling safe, efficient and reproducible administration that requires minimal clinical training. Introgen Therapeutics, Inc. 301 Congress Ave., Suite 1850 Austin, TX Telephone: Fax: c.burke@introgen.com Website: Booth Number: 202 Introgen Therapeutics, Inc. is a biopharmaceutical company focused on the discovery, development and commercialization of targeted molecular therapies or the treatment of cancer and other diseases. Introgen maintains integrated research, development, manufacturing, clinical and regulatory departments and operates multiple manufacturing facilities including a commercial scale cgmp manufacturing facility. Kinovate Life Sciences, Inc. 501 Via del Monte Oceanside CA, Telephone: Fax: Website: Booth Number: 114 Kinovate Life Sciences offers a range of novel in vitro gene delivery tools, including our unique biodegradable cationic polymer transfection reagent and revolutionary Fast Transfection Plates which drastically simplifies and speeds up the transfection assay process. We are also developing tools for sirna delivery to be launched later this year. Lentigen 1450 South Rolling Road, Suite Baltimore, MD Telephone: Fax: jessica.boehmer@lentigen.com Website: Booth Number: 216 Lentigen Corporation is focused on the manufacturing and development of lentiviral vectors in a wide range of applications. With its novel, highly efficient lentiviral vector system - LentiMaxTM, Lentigen plans to become the leading provider of lentiviral vector products and services for academic, government, biotechnology and pharmaceutical researchers. Mary Ann Liebert 140 Huguenot Street, 3rd Floor New Rochelle, NY Phone: Fax: info@liebertpub.com Website: Booth Number: 405 Mary Ann Liebert, Inc., publishers is known for establishing authoritative peer-reviewed journals in promising areas of science and biomedical research including Human Gene Therapy the definitive peer-reviewed journal covering all aspects of gene therapy. ( The Journal is a continuously updated directory of human gene marker and gene therapy clinical protocols. Complimentary copies of the Journal are available at the Mary Ann Liebert booth #

49 Exhibitor Descriptions Millipore Corporation 290 Concord Road Billerica, MA Telephone: Fax: Website: Booth Number: 308 Millipore s Bioscience Division provides innovative tools, services and biological reagents that drive advancements in biomedical and academic research as well as support the discovery and development of new pharmaceuticals. Our customers work in leading research laboratories across a variety of industries throughout the world. Millipore improves their laboratory productivity and efficiency through optimized workflows. Molecular Medicine BioServices, Inc Rutherford Road, Suite 200 Carlsbad, CA Phone: Fax: info@molecularmed.com Booth Number: 413 Molecular Medicine BioServices, Inc. provides enabling technologies for viral-based therapeutics. Located in Carlsbad, CA, the company is a leading contract manufacturing and clinical production resource for companies engaged in vaccine and gene therapy development. Molecular Medicine is committed to supporting clients from development through production and into clinical approval. Napa Jenomics Corporation Hibarigaoka-Kita Nishi-Tokyo Japan Telephone: Website: Booth Number: 401 NapaJen develops the predominant DDS technology to enables natural nucleic acid stably workable in vivo and sustain its primary function as originally-researched. The technology requires no chemical treatment but realizes stabilization of nucleic acid in vivo and passive targeting capability to antigen presenting cells. National Gene Vector Laboratories Indiana University Department of Medical and Molecular Genetics Medical Research & Library Building West Walnut Street Indianapolis, IN Telephone: Fax: lrubin@iupui.edu Website: Booth Number: 122 To continue our service to the gene therapy community the National Gene Vector Laboratories (NGVL) production centers have joined forces to form a consortium. We are able to offer ten years of experience in vector production as well as manufacture of other viral-based biologics. Our experience has allowed us to provide the highest quality vectors at affordable costs enabling development of early clinical studies by academic investigators. For further information please call the number above. Nature Publishing Group 75 Varick Street, Floor 9 New York, NY Telephone: g.lee@natureny.com Website: Booth Number: 215 Nature Publishing Group celebrates ASGT s 10 years of outstanding research in gene therapy as the new publisher of Molecular Therapy. The NPG portfolio combines the continued excellence of Nature and its associated research/review journals with over 35 leading academic journals and the Nature Clinical Practice series. Visit Stand no: 215 for free sample copies. Nature Technology Corporation 4701 Innovation Dr. Lincoln, NE Telephone: Fax: hodgson@natx.com Website: Booth Number: 407 Nature Technology Corporation (NTC) offers rational biologics development and manufacturing services, including vector design, strain engineering, fermentation, process development, downstream purification, and technology transfer. NTC s products include nucleic acid-based vaccines and therapeutics, plasmids, viral vectors, recombinant enzymes and proteins, production host strains and multipartite biopharmaceuticals. 48

50 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Exhibitor Descriptions NIH, Office of Biotechnology Activities 6705 Rockledge Drive, Suite 750 Bethesda, MD Telephone: Fax: bm128w@nih.gov Website: Booth Number: 206 The NIH Office of Biotechnology Activities (OBA) will be exhibiting its new initatives, and staff will be available to answer questions about the oversight of recombinant DNA research, including human gene transfer research. Meeting participants will be given handson demonstrations of OBA s Genetic Modification Clinical Research Information System (GeMCRIS). Northern Lipids Inc Northbrook Court Burnaby, B.C. V5J 5J1 Canada Telephone: Fax: vinaychhatre@northernlipids.com Website: Booth Number: 220 NLI is a Contract Manufacturing Organization that also specializes in offering services for pre-clinical & clinical development of lipidbased pharmaceutical formulations such as liposomes, emulsions and micelles. We provide LIPEX brand of Extruders, customized formulation development, scale up and analytical services from R & D through to commercialization. NovaMatrix/FMC BioPolymer PO Box 8 Princeton, NJ Telephone: Fax: frank_rauh@fmc.com Website: Booth Number: 108 NovaMatrix, a business unit of FMC Corporation, is a leading supplier of ultra-pure biomaterials for biomedical applications. Products include ultra-pure PRONOVA alginates, PROTASAN chitosans and hyaluronates. New products include NOVATACH peptide coupled alginates for tissue engineering and NOVAFECT special chitosans for gene delivery. All products are cgmp and ISO compliant. Penn Vector University of Pennsylvania Gene Therapy Program 125 S 31st Street Philadelphia, PA Telephone: Fax: aksandhu@mail.med.upenn.edu Website: Booth Number: 403 Penn Vector is a University of Pennsylvania Core laboratory that provides a broad range of services including production of viral vectors. The Core offers vectors based on adenoviruses, lentiviruses and adenoassociated viruses (AAV) and specializes in the production of novel vectors including those based on a variety of AAV serotypes. PolyPlus Transfection Bioparc BP 90018, Illkirch France Telephone: pbelguise@polyplus-transfection.com Booth Number: Polyplus-transfection is a Life Science company dedicated to research, development and marketing of transfection reagents. Polyplus provides state-of-the-art high quality reagents for in vitro and in vivo DNA gene delivery including cgmp grade. Our porfolio includes reagents for sirna delivery, protein delivery in live cells and protein production in synthetic medium. Puresyn, Inc. 87 Great Valley Parkway Malvern, PA Telephone: Fax: leslie@puresyn.com Website: Booth Number: 102 Puresyn, Inc., a Separations and Purification Technology Company, developing, manufacturing and marketing state-of-the-art products and services for nucleic acids and other biological molecules utilizing our proprietary polymer technology, PolyFlo for production and purification of gene delivery products including plasmids and recombinant viruses at scales and customized specifications. 49

51 Exhibitor Descriptions Rich-Mar PO Box 879 Inola, OK Telephone: Fax: Website: Booth Number: 204 Rich-Mar developed the first Sonoporation device in 1997 and since been customizing devices for drug delivery and gene therapy. Rich-Mar is involved in clinical trials in Europe and the US. Sonitron parameters can be customized to meet specific requirements. The Sonitron has been successful in-vitro, in-vivo and in-ovo. Sartorius Biotech Inc. 131 Heartland Blvd. Edgewood, NY Telephone: Fax: Website: Booth Number: 212 Sartorius provides a complete line of laboratory products from drug discovery to quality control. As a total solution provider, we offer products for all your laboratory needs including filtration, purification, cell culture, microbiology, and lab water systems. Stop by our booth, and learn more about Sartorius AdenoPACK adenovirus purification kits. SeqWright, Inc W. Bellfort, Suite 2001 Houston, TX Telephone: Fax: Website: Booth Number: 203 SeqWright is a GLP molecular biology service company specializing in Adenoviral DNA Sequencing, Affymetrix Microarray, Quantitative PCR Analysis, Custom Cloning and Mutagenisis, Microbial ID, and sirna Vector Construction. Southern Research Institute 2000 Ninth Avenue South Birmingham, AL Telephone: Fax: jefferson@sri.org Website: Booth Number: 311 Southern Research is a science-driven, client-focused contract research organization involved in drug discovery and nonclinical drug development. Extensive experience in gene therapy product development (vector construction, efficacy evaluations, nonclinical toxicology, and biodistribution studies with Q-PCR One of two National Toxicology Centers designated by the National Gene Vector Laboratories Spectrum Laboratories Broadwick St. Rancho Dominquez, CA Telephone: Fax: badamson@spectrumlabs.com Website: Booth Number: 320 Spectrum Laboratories manufactures a complete line of separation products for the biotech, pharmaceutical and diagnostic industries. Spectrum s filtration products include the largest selection of disposable hollow fiber membranes and a full scale range of disposable upstream and downstream Tangential Flow Filtration Systems. St. Jude Children s Research Hospital 332 N. Lauderdale, MS 304 Memphis, TN Telephone: (901) Fax: (901) jacaranda.vanrheenen@stjude.org Website: Booth Number: 201 St. Jude Children s Research Hospital is a non-profit biomedical research institution in Memphis, TN, where 180 basic science and clinical researchers investigate the molecular basis of both normal cellular and diseased processes. Visit our booth to discuss and apply for Postdoctoral Fellowship positions for PhD, MD and PharmD students. 50

52 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Exhibitor Descriptions Vandalia Research Inc 940 Fourth Avenue, Suite 350 Huntington, WV Telephone: Fax: gregg@vandaliaresearch.com Website: Booth Number: 319 Vandalia Research, Inc. provides large-scale DNA production services using its patent-pending Triathlon system, which enables the in vitro production of milligram and gram quantities of specific, high-quality dsdna sequences with the polymerase chain reaction. Vector Gene Technology Co. Ltd Contact Person: David Yang 6 Yongchangzhong Rd BDA Beijing 92129, China Telephone: X110 david_y@agtc.com.cn Website: Booth Number: 106 VGTC is a leading contract manufacturer in China for gene therapy vectors providing vector construction, cell line development, analytical method development, cell banking and non-gmp and cgmp manufacturing services for adenovirus, AAV and plasmid DNA vectors with high quality and very competitive price. Visualsonics 3080 Yonge Street, Suite 6100 Toronto, ON M4N 3N1 Canada Telephone: Fax: svanwyk@visualsonics.com Website: Booth Number: 306 VisualSonics Vevo 770 high-resolution in vivo micro-imaging is a novel syetmto visualize and measure physiological and anatomical phenomena in small animals from E5.5 through to adulthood noninvasively in real-time. The Vevo 770 enables in vivo assessment of anatomical structures, hemodynamic function, molecular biomarkers and guided injection of stems cells and genetic materials in longitudinal studies of small animals. Waisman Clinical Biomanufacturing Facility 1500 Highland Ave Madison, WI Telephone: keach@waisman.wisc.edu Website: Booth Number: 411 The Waisman Clinical Biomanufacturing Facility (WCBF) is a dedicated manufacturing facility that provides cgmp manufacturing services for producing biotherapeutics for early-stage human clinical trials. Services that are provided by the WCBF range from process development to clinical production and product fill and finish, all with full quality system support. 51

53 Exhibitor Information General Information The Exhibit Hall is located in Hall 4AB, near the South Lobby of the Washington State Convention & Trade Center. All participants are urged to allow adequate time in your daily schedule to visit the exhibits. The companies, organizations and institutions exhibiting at the ASGT Annual Meeting will be providing you with the latest information on products and services directly related to your professional needs, as well as recruiting for open positions. Exhibit Hall Hours Exhibits will be open Wednesday, May 30 through Friday, June 1, Exhibit Installation: Wednesday, May 30 Hall Hours: Wednesday, May 30 Thursday, May 31 Friday, June 1 Friday, June 1 Exhibit Dismantlement: Friday, June 1 9:00 am 4:00 pm 5:00 pm 9:00 pm 5:00 pm 8:00 pm 12:00 pm 2:00 pm 5:15 pm 8:15 pm 8:15 pm Midnight Note: All exhibit booths must be staffed during hours. Packing or equipment, literature of dismantling of exhibits will not be permitted until after 8:15 pm on Friday, June 1, Exhibitor Registration The Exhibitor Registration desk is located in the South Lobby of the Washington State Convention & Trade Center, across from Exhibit Hall 4AB. Exhibitor Registration Hours Wednesday, May 31 Thursday, June 1 Friday, June 2 9:00 am 7:00 pm 7:00 am 5:00 pm 11:00 am 5:00 pm Exhibitor Badge Policy Admission to the Exhibit Hall will be by name badge only. Security guards will monitor exhibit hall entrances for proper I.D. Exhibit booth personnel must show an official ASGT exhibitor name badge in order to gain access to the exhibit hall during installation, show, or dismantlement hours. Independent contractor personnel, hired by an exhibitor to install and dismantle their display, do not need to show exhibitor badges, but should possess identification that they are employed by the independent contractor. If independent contractors do not possess proper identification, they should obtain a name badge at the on-site exhibitor registration desk. Endorsement Disclaimer Products and services displayed in the Exhibit Hall or advertised in this program occur by contractual business arrangements between the ASGT and participating companies and organizations. These arrangements do not constitute or imply an endorsement by the ASGT of these products and services. Seattle Fun Facts Seattle is a hub for the Pacific Northwest and is a crossroads for travel and world trade. The Seattle-Tacoma (Sea-Tac) International Airport which serves the city is close to the downtown area, being only a minute drive. Located in the center of the Puget Sound region, Seattle sits between Elliott Bay and Lake Washington, and is surrounded by water and mountains. The city s attraction lies in the stunning views seen from many vantage points throughout its neighborhoods. The height of Mount Rainier is truly appreciated when seen from within the city, as it puts skyscrapers to shame from 200 miles away. Seattle Center & the Space Needle The Seattle Center was built as the United States Science Pavilion for the Seattle World s Fair of This complex is home to the Seattle Opera, Seattle Repertory, and the Pacific Northwest Ballet to name a few. The center is also home to The Space Needle, which opened on the first day of the World Fair, and features an observation deck, restaurant and cocktail bar within its Jetsons style top. This most recognizable landmark in Seattle can be reached by a 90-second monorail ride from downtown s Westlake shopping center. Pike Place Market Situated just above the waterfront, the market is popular with natives and tourists alike. A vital part of Seattle s economy, the market features 600 businesses, including farmers and merchants offering fresh vegetables, seafood, food bars, cafes, restaurants, crafts, art work, and gifts. Home of the original Starbucks, the market was born in 1907 and is the oldest continually operating farmers market in the USA. Seattle Aquarium Walk under the water in a glass dome as bluntnose sixgill sharks and other Elliott Bay creatures swim all around you. Pioneer Square Saved from the wrecking ball by popular outcry, Seattle s oldest neighborhood is a 17-square-block National Historic District. Its charming, red-brick buildings have been revived and now hold a great arts, café, shopping, and nightlife scene. The Seattle Waterfront The waterfront is a bustling collection of attractions, restaurants and shopping. It is also the starting point for ferries and cruise ships, including the Victoria Clipper and Argosy boat tours which are located here. Feed the seagulls, stroll by the wooden piers of Waterfront Park, admire the view or shop for souvenirs. 52

Final Program Housing Information ❶ Sheraton Seattle Headquarters Hotel 1400 Sixth Avenue Seattle,

com/sheraton/seattle ❷ Grand Hyatt Seattle 721 Pine Street Seattle, Washington 98101 Phone: (206)

com ❸ The Mayflower Park Hotel 405 Olive Way Seattle, Washington 98101 Phone: (206) 623-8700

54 Final Program Housing Information ❶ Sheraton Seattle Headquarters Hotel 1400 Sixth Avenue Seattle, Washington Phone: (206) ❷ Grand Hyatt Seattle 721 Pine Street Seattle, Washington Phone: (206) ❸ The Mayflower Park Hotel 405 Olive Way Seattle, Washington Phone: (206) ❹ The Westin Seattle th Avenue Seattle, Washington Phone: (206) ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007

55 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Oral Abstract Session 240 AAV Vector Integration and Immunology 3:00 pm 5:00 pm Room 4C1/4C2 1 3:00 PM AAV Vector Integration at a Common Locus Leads to Hepatocellular Carcinoma in Mice Daniel G. Miller, Anthony Donsante, Yi Li, Carole Vogler, Elizabeth M. Brunt, David W. Russell, Mark S. Sands. 2 3:15 PM AAV Vectors Do Not Increase the Risk of Tumor Formation in p53 Deficient Models Joerg Schuettrumpf, Stefano Baila, Fayaz Khazi, Jianhua Liu, Ralph Bunte, Valder R. Arruda. 3 3:30 PM Loss of Newly Formed Double-Stranded Genomes Is the Primary Cause of raav Low Transduction Efficacy Jinhui Wang, Jing Xie, Hui Lu, Lingxia Chen, Bernd Hauck, Richard Jude Samulski, Weidong Xiao. 4 3:45 PM AAV Labeling of Break-Prone Palindromes in Mammalian Genomes: Exploitation of the In Vivo raav Biology for Studies of Genomic Instability in Living Animals Katsuya Inagaki, Susanna M. Lewis, Xiaolin Wu, Congrong Ma, David Munroe, Sally Fuess, Theresa A. Storm, Mark A. Kay, Hiroyuki Nakai. 5 4:00 PM Site-Specific Integration of AAV2/2 and AAV2/8 Vectors in Hepatocytes In Vivo Zhongya Wang, Terry Storm, Raymond Monnat, Jr, Milton Finegold, Mark Kay, Markus Grompe. 6 4:15 PM The Mre11/Rad50/Nbs1 Complex Restricts raav Transduction by Limiting Single-Stranded to Double- Stranded DNA Genome Conversion Jose Alejandro Palacios, Miguel Mano, Tiziana Cervelli, Lorena Zentilin, Rachel Schwartz, Matthew D. Weitzman, Mauro Giacca. 7 4:30 PM Slow Entry Process Results in Adeno-Associated Virus Sensitivity to Antibody Mediated Neutralization Samuel L. Murphy, Hojun Li, Alexander Schlacterman, Katherine A. High. 8 4:45 PM Extensive AAV Epidemiological Study Identifies a Novel AAV Variant with Low Seroprevalence Roberto Calcedo, Luk H. Vandenberghe, Jianping Lin, Guang-Ping Gao, Maria Limberis, Donald Siegel, Matthew Cleveland, Alberto Auricchio, Matthew Price, Nicholas Cleveland, Laurette Steenssens, Jo Backhouse, Myrsini Moschou-Parara, James M. Wilson. Oral Abstract Session 241 Adenovirus Vectors: Immunity and Toxicology 3:00 pm 5:00 pm Room :00 PM Adenovirus Vector Induced Immune Responses Are Dependent upon Multi-Faceted Interactions with Proteins of Both the Alternative and Classical Complement Pathways In Vivo Jeannine Scott, Anne Kiang, Daniel Appledorn, Aaron McBride, Andrea Amalfitano. 10 3:15 PM Fully Detargeted Polyethylene Glycol-Stealthed Adenovirus Vectors Demonstrate High Potency for Genetic Vaccination and Are Able To Escape from Pre-Existing Anti-Ad Humoral Immunity Andreas Wortmann, Sabine Vöhringer, Tatjana Engler, Stefan Kochanek, Florian Kreppel. 11 3:30 PM Characterization of Immune Responses Provoked by Alternative Adenovirus Serotypes Spanning Groups A-E Daniel M. Appledorn, Jeannine M. Scott, Anne Kiang, Andrea Amalfitano. 12 3:45 PM Adenovirus Membrane-Lytic Activity Is Required for Kupffer Cell Toxicity and Contributes to the Innate Cytokine Response Jeffrey S. Smith, Zhili Xu, Jie Tian, Andrew P. Byrnes. 13 4:00 PM Toxicology of PEGylated Helper-Dependent Adenovirus in Non-Human Primates Piyanuch Wonganan, Eleonora Leggiero, Kathy Brasky, Joe Dekker, Dalila Astone, Lucio Pastore, Maria A. Croyle. 14 4:15 PM Adenovirus pix Gene Is Co-Expressed with Transgene Expression and May Induce Host Immune Responses in First-Generation Adenovirus Vectors Michio Nakai, Kazuo Komiya, Masashi Murata, Toru Kimura, Masaharu Kanaoka, Yumi Kanegae, Izumu Saito. 15 4:30 PM Engineering a Recombinant Adenovirus for Targeted Transduction of Neutrophils for Use as Anti-Inflammatory Cell Vehicles Justin C. Roth, Michael O. Alberti, Larisa Pereboeva, Svetlana Komarova, Stanton L. Gerson, David T. Curiel. 16 4:45 PM Modification of Ad5 Hexon Hypervariable Regions Circumvents Pre-Existing Ad5 Neutralizing Antibodies Joseph T. Bruder, Elena Semenova, Charlie Thomas, Noelle B. Patterson, Maureen E. Stefaniak, Keith Limbach, C. R. King, Denise L. Doolan. 54

56 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Oral Abstract Session 242 Physical Methods of Delivery 3:00 pm 5:00 pm Room 4C3 17 3:00 PM GnRH-Expressing Plasmid Delivered by Constant-Current Electroporation Can Regulate the Reproductive Axis in Horses William A. Storer, Donald F. Thompson, Jr, Kenneth Bondioli, Amir S. Khan, Patricia A. Brown, Ruxandra Draghia-Akli. 18 3:15 PM Non-Viral Cutaneous Gene Transfer DNA Using In Vivo Electroporation Richard Heller, Mark J. Jaroszeski, Yolmari Cruz, Amy Donate, Bernadette Ferraro, Richard Gilbert, Kenneth Ugen, Loree C. Heller. 19 3:30 PM Optimised Pulses for Electro Gene Transfer to Muscle Tissue Reduce Adverse Effects on Cellular Integrity and Function Hanne Gissel, Pernille H. Moller, Julie Gehl. 20 3:45 PM Optimization of Definity Microbubble Concentrations and Acoustic Pressure Increases Ultrasound-Mediated Gene Transfer Efficiency While Reduces Tissue Damage Zhenping Shen, Andrew A. Brayman, Liping Chen, Baowei Peng, Carol H. Miao. 21 4:00 PM Copolymer Protected BMP-2 Gene Vectors Immobilized in Poly(D,L-Lactide) Films on Titanium Implants Mediate Bone Regeneration in a Rat Mandibular Critical Bone Defect Model Andreas Kolk, Cornelia Haczek, Christoph Pautke, Martin Kullmer, Herbert Deppe, Andreas Neff, Axel Stemberger, Christian Plank. 22 4:15 PM Development of Computer-Controlled Injection Device for Hydrodynamic Gene Delivery Takeshi Suda, Dexi Liu. 23 4:30 PM Efficient Delivery and Long-Term Expression of a 135 kb LDLR Genomic DNA Locus In Vivo Following Hydrodynamic Tail Vein Injection Olivia C. Hibbitt, Richard P. Harbottle, Simon N. Waddington, Christine A. Bursill, Charles Coutelle, Keith M. Channon, Richard Wade-Martins. 24 4:45 PM Comparative Gene Expression In Vivo after Hydrodynamic Delivery and Polyethyleneimine-Conjugated Delivery of DNA Using the Sleeping Beauty Transposon System Kelly M. Podetz-Pedersen, Jason B. Bell, R. Scott McIvor, Perry B. Hackett. Oral Abstract Session 243 Neurologic I 3:00 pm 5:00 pm Room :00 PM The Role of Alpha-Synuclein (α-syn) Phosphorylation at Ser-129 on the Development of Parkinson-Like Neurodegeneration Oleg Gorbatyuk, Shaudong Li, Fredric P. Mandfredsson, Frederic M. Nguyen, Layla Sullivan, Weijun Chen, Galina Kondrikova, Craig A. Meyers, Ronald J. Mandel, Nicholas Muzyczka. 26 3:15 PM Correction of Brain Lesions in MPS I Dogs after Neonatal Systemic Gene Therapy May Involve Migration of Blood Cells into the Brain Mark E. Haskins, Anne M. Traas, Ping Wang, Xiucui Ma, Mindy Tittiger, Patricia O Donnell, Charles Vite, Katherine P. Ponder. 27 3:30 PM Analysis of the Transduction and Biodistribution Patterns of Alternative AAV Vector Serotypes in the CNS of Adult Rhesus Macaques Bruce A. Bunnell, Jason Dufour, Mette Flaat, Jeanne Fisher, Xavier Alvarez, Kate Baker, Pete Didier, Andrew Lackner, John H. Wolfe. 28 3:45 PM Targeted Delivery of Glycine Receptors to Peripheral Neurons as Treatment for Pain Michael Cascio, James Goss, David Krisky, Rahul Scrinivasin, Joseph Glorioso. 29 4:00 PM Genetic Engineering of Neural Stem Cells Specifies Motor Neuron and Oligodendrocyte Differentiation in the Adult Spinal Cord Delphine Bohl, Song Liu, Stéphane Blanchard, Michäel Hocquemiller, Georg Haase, Jean-Michel Heard. 30 4:15 PM Neuronal Specificity of HSV/Sleeping Beauty Amplicon Transduction In Utero Is Driven Primarily by Tropism and Cellular Co-Factor Expression Profiles during Embryogenesis Elise B. Peterson, Michael A. Mastrangelo, Howard J. Federoff, William J. Bowers. 31 4:30 PM AAV Mediated Gene Transfer of IGF-1 and VEGF to the Ventricular System Provides Significant Therapeutic Benefit in a Mouse Model of Amyotrophic Lateral Sclerosis James C. Dodge, Christopher Treleaven, Jennifer Clarke, Wendy Yang, Heather Martin, Chalonda Handy, Mark Hester, Tatyana V. Taksir, Denise A. Griffiths, Seng H. Cheng, Brian K. Kaspar, Lamya S. Shihabuddin. THURSDAY, MAY 31 55

57 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Oral Abstract Session 243 continued Neurologic I 3:00 pm 5:00 pm Room :45 PM HSV-Mediated Delivery of a TrkA-Repressing Engineered Zinc Finger Protein Transcription Factor Reduces Nociception in a Mouse Model of Bone Cancer Pain James R. Goss, H. Steve Zhang, David M. Krisky, Andrew R. McNamara, Yann Jouvenot, Siyuan Tan, Shaohua Huang, James B. Wechuck, Darren Wolfe, S. Kaye Spratt, Joseph C. Glorioso, Philip D. Gregory. Oral Abstract Session 244 Cancer Immunotherapy: Dendritic Cells, Vaccines and Immune Modulation 3:00 pm 5:00 pm Room 608/ :00 PM CD40-Targeted Adenoviral Vector Transduction of Dendritic Cells Disha Mody, Don A. Sibley, Yoshinobu Odaka, Jagat Poduturri, Xiao L. Li, David T. Curiel, Alexander Pereboev, J. Michael Mathis. 34 3:15 PM Intratumoral Manipulation of Dendritic Cells Elicits Immunological Memory in a Recurrent, Syngeneic Glioma Model Gwendalyn D. King, James F. Curtin, A. K. M. Ghulam Muhammad, Carlos Barcia, Daniel Larocque, Marianela Candolfi, Chunyan Liu, Sarah B. Honig, Nico VanRooijen, Antoni Ribas, Kurt M. Kroeger, Regis Josien, Pedro R. Lowenstein, Maria G. Castro. 35 3:30 PM Connective Tissue Growth Factor Encoding Tumor Antigen Generates Potent Antigen-Specific Cancer Immunotherapy through Prolonging Life-Span of Antigen-Presenting Cells Wen-Fang Cheng, Chi-An Chen, Chang-Yao Hsieh, Ming-Cheng Chang. 36 3:45 PM Combination of Tumor-Site Located CTLA-4 Blockade and Systemic Regulatory T Cell Depletion Induces Anti-Tumor Immunity Sebastian Tuve, Bing-Mae Chen, Ying Liu, Tian-Lu Cheng, Shaoheng Ni, Zong-Yi Li, Steve R. Roffler, Andre Lieber. 37 4:00 PM In Vivo Targeting of Tumor Antigens to DCs by Administration of Transduced Lymphocytes Results in Robust and Prolonged Immunization Cristina Rainelli, Laura Raccosta, Arcadi Cipponi, Raffaella C. Fontana, Daniela Maggioni, Fabio Ciceri, Marco Bregni, Claudio Bordignon, Catia Traversari, Vincenzo Russo. 38 4:15 PM Co-Expression of Cytokine and Suicide Genes To Enhance the Activity and Safety of Tumor Specific Cytotoxic T Lymphocytes Juan F. Vera, Concetta Quintarelli, Barbara Savoldo, Greta M. P. Giordano Attianese, Aaron Foster, Martin Pule, Helen E. Heslop, Cliona M. Rooney, Malcolm K. Brenner, Gianpietro Dotti. 39 4:30 PM CD3/CD28 Activation and Culture with IL-7 and IL-15 Permit Efficient Transfer of a Suicide Gene in Highly Alloreactive Central Memory Lymphocytes Sara Mastaglio, Shin Kaneko, Attilio Bondanza, Maurilio Ponzoni, Luca Aldrighetti, Salvatore Toma, Marina Radrizzani, Simona La Seta, Fabio Ciceri, Claudio Bordignon, Chiara Bonini. 40 4:45 PM Generating Cytotoxic T Lymphocytes Against Prostate Specific Membrane Antigen by AAV-Loaded Dendritic Cells for the Treatment of Prostate Cancer Mahendran Mahadevan, Yong Liu, Changxuan You, Rongcheng Luo, Paul L. Hermonat. Oral Abstract Session 245 Cancer: Apoptosis & Suicide 3:00 pm 5:00 pm Room 606/ :00 PM Human Neural Progenitor Cells Homing to Glioma Xenografts Zahidul Khan, Mette Willer, Zoran Gojkovic, Bo S. Jensen, Bengt Juliusson, Tomas J. Ekstrom, Per M. Almqvist. 42 3:15 PM Drug-Virus Interaction: Effect of Administration of Recombinant Adenoviruses on the Pharmacokinetics of Docetaxel in a Rat Model Piyanuch Wonganan, William C. Zamboni, Sandy Strychor, Joe D. Dekker, Blake Roessler, Brian Sidone, Maria A. Croyle. 43 3:30 PM Nanoparticle-Delivery of Diphtheria Toxin DNA Inhibits Ovarian Tumor Growth Janet A. Sawicki, Yu-Hung Huang, Weidan Peng, Daniel G. Anderson, Robert Langer. 44 3:45 PM Phase I Study of Ad5-TRAIL in Men with Clinically Organ Confined Prostate Cancer Thomas S. Griffith, Badrinath R. Konety, Fadi N. Joudi, Howard Aubert, Michael B. Cohen, Timothy L. Ratliff, Richard D. Williams. 56

58 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Oral Abstract Session 245 continued Cancer: Apoptosis & Suicide 3:00 pm 5:00 pm Room 606/ :00 PM Prognostic Determinants Associated with Efficacy of Adenoviral p53 Gene Therapy (Advexin) in Patients with Recurrent Squamous Cell Carcinoma of the Head and Neck (SCCHN) J. J. Nemunaitis, C. Bier-Laning, G. L. Clayman, D. VanEcho, L. Guertin, J. Hamm, R. Dreicer, G. H. Yoo, H. Minn, M. Bekradda, W. Sutherland, K. B. Menander, R. E. Sobol, W. J. Goodwin, Advexin H&N Study Group. 46 4:15 PM Adenoviral-Mediated Gene Transfer of Single Chain Fv 425:sTRAIL Fusion Protein Induces Target Cell Restricted Apoptosis in EGFR-Positive Tumor Cells and a Potent Bystander Effect without Toxicity In Vivo Hidde J. Haisma, Marike H. Dijkstra, Edwin Bremer, Wijnand Helfrich. 47 4:30 PM Heat Shock Protein Inhibitors Increase the Efficacy of Measles Virotherapy Chunsheng Liu, Charles Erlichman, Cari J. McDonald, James N. Ingle, Paula Zollman, Ianko Iankov, Stephen J. Russell, Evanthia Galanis. 48 4:45 PM Increased Potency of Engineered FasL-Expressing T Cells Is Modified in Primary Prostate Cancer Cells by Common Chemotherapeutic Agents Julie Symes, Neil Fleshner, Daniel H. Fowler, Jeffrey A. Medin. Oral Abstract Session 246 Oncolytic Viruses 3:00 pm 5:00 pm Room :00 PM Exponential Enhancement of Oncolytic VSV Potency by Vector-Mediated Suppression of Anti-Viral Inflammatory Responses In Vivo Lan Wu, Jennifer Altomonte, Li Chen, Marcia Meseck, Oliver Ebert, Adolfo García-Sastre, John T. Fallon, Savio L. C. Woo. 50 3:15 PM Phase I Clinical Trial Study Ultilizing Oncolytic Adenovirus with HSP Gene Expression (H103) in Treating Patients with Advanced Solid Tumor Hongli Liu, Yu Wang, Yong Ben, Datong Chu, Fang Hu. 51 3:30 PM IL13 Displaying Retargeted Oncolytic Measles Virus (MV) Strains Have Significant Activity and Improved Therapeutic Index Against Gliomas Cory Allen, Georgia Paraskevakou, Ianko Iankov, Mark Schroeder, Raj K. Puri, Stephen J. Russell, Evanthia Galanis. 52 3:45 PM Targeting Tissue Tropism of Coxsackievirus A21, an Oncolytic Enterovirus Elizabeth Kelly, Elizabeth Hadac, Stephen J. Russell. 53 4:00 PM Human MSC for Delivery of Infectivity Enhanced Oncolytic Adenoviruses into Orthotopic Lung and Breast Tumors T. Hakkarainen, M. Särkioja, P. Lehenkari, S. Miettinen, T. Ylikomi, R. A. Desmond, A. Kanerva, A. Hemminki. 54 4:15 PM Mechanisms of Resistance of Primary Ovarian Cancer Cells to Viral Oncolysis Robert Strauss, Pavel Sova, Ying Liu, ZongYi Li, David Pritchard, Nicole Urban, Charles Drescher, Nancy Kiviat, Andre Lieber. 55 4:30 PM Design of ICP0 Deficient HSV Oncolytic Vectors for Treatment of Glioblastoma Paola Grandi, Adriana Forero, A. Niranjan, Wendy Fellows, Hideho Okada, David Klinkenberg, David Krisky, Joseph Glorioso. 56 4:45 PM Prostate Restricted Replicative Adenovirus Expressing Human Endostatin-Angiostatin Fusion Gene Exhibiting Dramatic Antitumor Efficacy Xiong Li, You-Hong Liu, Sang-Jin Lee, Thomas A. Gardner, Meei-Huey Jeng, Chinghai Kao. Oral Abstract Session 247 Oligonucleotide Therapies I 3:00 pm 5:00 pm Room 4C4 57 3:00 PM Viral Delivery of shrnas as a Potential Therapy for Huntington s Disease Jodi L. McBride, Scott Q. Harper, Patrick D. Staber, Ines Martins, Haim Burstein, Richard W. Peluso, Barry Polisky, Barrie Carter, Beverly L. Davidson. 58 3:15 PM Optimization of Zinc Finger Nucleases for Gene Targeting in Mammalian Cells Shondra M. Pruett, Jon P. Connelly, Morgan Maeder, J. Keith Joung, Matthew H. Porteus. 59 3:30 PM Improved Toxicity Profile of Pol II- over Pol III-Promoted Hairpin RNAs Due to Reduced Expression Levels Jeffery C. Giering, Dirk Grimm, Theresa A. Storm, Hui Xu, Mark A. Kay. 60 3:45 PM Transfer of sirna Molecules Via Gap Junctions in Heart Cells Eddy Kizana, Eugenio Cingolani, Genaro A. Ramirez-Correa, Eduardo Marban. THURSDAY, MAY 31 57

59 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Oral Abstract Session 247 continued Oligonucleotide Therapies I 3:00 pm 5:00 pm Room 4C4 61 4:00 PM Supplementation with hrad51/54 or Antibodies Against Ku70/86 Increase the Efficiency of Single Stranded DNA- Mediated Genome Alteration in Preimplantation Stage Mouse Embryos Viktor Morozov, Eric F. Wawrousek. 62 4:15 PM Deciphering microrna Function in the CNS Gumei Liu, Ines Martins, Beverly L. Davidson. 63 4:30 PM Antisense Oligonucleotide Induced Exon Skipping as a Therapy for Duchenne Muscular Dystrophy Steve D. Wilton, Abbie A. Fall, Penny M. Harding, Patrick Iversen, Susan Fletcher. 64 4:45 PM SFHR Gene Targeting Strategy Optimization: Useful Tools and Increased Efficiency of Repair A. Filareto, M. Sarra, A. Malgieri, E. Bruscia, D. Fina, P. Spitalieri, G. Monteleone, M. Sanchez, G. Novelli, F. Sangiuolo. Poster Session I 5:00 pm 8:00 pm Hall 4AB Integration and Genotoxicity 65 Karyotypic Changes May Be Inevitable in Retroviral Gene Therapy Jaehyung Yoon, Vivian Yip, Sally Hwang, Lillian Yun, Youngsuk Yi, Moon Jong Noh, Kwan Hee Lee. 66 Comparison of HIV-Derived Lentiviral and MLV-Based Gammaretroviral Vector Integration Sites in Primate Repopulating Cells Brian C. Beard, David Dickerson, Kate Beebe, Christina Gooch, James Fletcher, Tulin Okbinoglu, Mike A. Jacobs, Rajinder Kaul, Hans-Peter Kiem, Grant D. Trobridge. 67 Multiple Tandem Insulators May Reduce Retroviral Insertional Activation of Oncogenes and Lymphomagenesis Koyel Mitra, Mei Su, Jack Lenz. 68 Improving the Safety of HIV-1 Derived Gene Therapy Vectors: Reducing the Rate of SIN Repair and Polyadenylation Signal Readthrough Rachel Koldej, Donald S. Anson. 69 Site-Specific Genomic Integration of Lentiviral DNA Circles Brian Moldt, Nicklas H. Staunstrup, Maria Jakobsen, Jacob G. Mikkelsen. 70 Assessing Gammaretroviral Vector Integration Preferences for DNase Hypersensitive Sites Mingdong Liu, Peter J. Sabo, Michael S. Kuehn, John A. Stamatoyannopoulos, David W. Emery. 71 Determination of the Profile and Site Preference of Lentiviral Vector Integration In Vivo and in Primary Astrocyte Cultures Caroline V. Hacker, Yohann D. Moal, Gareth S. Ralph, Susan M. Kingsman, Kyriacos A. Mitrophanous, James E. Miskin. 72 Retroviral Integration Sites in Mucopolysaccharidosis VII Dogs Thomas M. O Malley, Paula S. Henthorn, Katherine P. Ponder, Mark E. Haskins. 73 Identification of Genetic Insulator Elements for Safe Gene Therapy Viral Integrating Vectors Cecile Bauche, Armelle Gaussin, Julien De Royer, Jean Francois Mouscadet, Christian Auclair, Nicholas Mermod, Odile Y. Cohen- Haguenauer. 74 Making Retroviral Gene Therapy Safer: Prevention of Retrovirus-Mediated Activation of Cellular Genes near the Integration Site by Engineering of the LTR Youngtae Hong, Nam-Kyung Yoon, Sujeong Kim, Sunyoung Kim, Joong Gon Kim, Jung Woo Rhim, Hyoung Jin Kang, Karim Lee, Jiwon Jang. AAV Production and Modification 75 Targeting of Angiogenic Endothelial Cells Using a New AAV- 2 Insertion Site Jorge Boucas, Kerstin Lux, Anke Huber, Sibille Humme, Michael Hallek, Luca Perabo, Hildegard Buning. 76 In Vivo Selection of Targeted Gene Transfer Vectors from AAV Random Peptide Display Libraries Ying Ying, Mueller Oliver, Waterkamp Daniel, Kleinschmidt A. Juergen. 77 Genetic Modification of AAV1 Capsid for MN Targeted Gene Delivery Thais Federici, Adam Davis, Qingshan Teng, Jonathan Riley, Jeffrey S. Bartlett, Nicholas M. Boulis. 78 Site Specific Modification of AAV Vector Particles with Biophysical Probes and Targeting Ligands Using Biotin Ligase Matthew D. Stachler, Irwin Chen, Alice Y. Ting, Jeffrey S. Bartlett. 58

60 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB AAV Production and Modification continued 79 Integration and Genotoxicity Making AAV Chimeras by STEP and Shuffle Peter Ward, Christopher E. Walsh. 80 Directed Evolution of Adeno-Associated Viral Vectors with Novel Cellular Gene Delivery Properties James T. Koerber, David V. Schaffer. 81 Directed Evolution of Adeno-Associated Virus in Primary Human Cells Ryan L. Jensen, Bruce C. Schnepp, Li Xu, Robert G. Whalen, K. Reed Clark, Philip R. Johnson. 82 Production of Infectious AAV Vectors in Insect Cells Using Novel Designed Rep and Cap Expression Cassettes Haifeng Chen. 83 Acceleration and Augmentation of AAV-Mediated Gene Expression David D. Dickey, Katherine J. D. A. Excoffon, Joseph Zabner. 84 High Titer Adeno-Associated Viral Vector Production by a Vero-Derived Producer Cell Line Clifford J. Beall, Philip R. Johnson, K. Reed Clark. 85 Insect Cell Production System: Alternative Startcodon Use for Improved AAV Infectivity Andrew Bakker, Masashi Urabe, Saskia Haast, Janneke Meulenberg, Jaap Twisk, Lizzy Comijn, Keiya Ozawa, Sander van Deventer, Wim Hermens. 86 Generation of More Infectious Type 1 AAV Vectors in Insect Cells Masashi Urabe, Hiroaki Mizukami, Andrew Bakker, Akihiro Kume, Wim Hermens, Keiya Ozawa. 87 Development and Characterization of a Relative Potency Assay for raav1 Vectors for Use as Vaccines Eric J. Pastor, Daryn J. Debelak, Christopher J. Gerard, Richard Peluso. 88 Equal In Vivo Efficacy of Recombinant AAV1 Vector Produced in Either Mammalian or Insect Cells Jaap Twisk, Colin J. D. Ross, Lizzy Comijn, Dennis Biesmans, Saskia Haast, Andrew Bakker, Edwin S. van Amersfoort, Sander J. H. van Deventer, Janneke J. M. Meulenberg, Michael R. Hayden, Wim T. Hermens. 89 Process Strategies for High Yield Production of Adeno- Associated Viruses in Insect Cells Marc G. Aucoin, Michel Perrier, Amine A. Kamen. 90 Scalable Method for Purification of Recombinant Adeno- Associated Viral Vector (raav) Type 6 Jingmin Zhou, Bern Hauck, J. Fraser Wright, Katherine A. High, Guang Qu. 91 Weak Anion Exchange Column Chromatography Enhances the Resolution of Separation of AAV Empty Capsid and Full Vectors Jingmin Zhou, Bern Hauck, J. Fraser Wright, Katherine A. High, Guang Qu. 92 Affinity Chromatography Purification of raav Vectors Produced by Transfection or rhsv Co-Infection Using CaptureSelect AAV Ligand Lijun Wang, David Knop. Inborn Errors of Metabolism I Storage Diseases Plus Others 93 Long-Term CNS and Somatic Correction in MPS II Mice after Intrathecal Delivery of AAV 9 Vector Lu Kang, Joseph Muenzer, Brian C. Eggert. 94 Systemic Administration of AAV9-SUMF1 Gene Results in Re-Activation of Sulfatases in a Mouse Model of Multiple Sulfatase Deficiency (MSD) Enrico Maria Surace, Carmine Spampanato, Carmine Settembre, Andrea Ballabio. 95 Targeting of Refractory Sites in Mucopolysaccharidosis Type I Using a Nonviral Transferrin-α-L-Iduronidase Fusion Gene Product Mark J. Osborn, Jakub Tolar, Bruce R. Blazar. 96 Insulin-Like Growth Factor-1 Rescues Galactocerebrosidase-Mediated Toxicity in Hematopoietic Stem Cells: Relevance for Gene Therapy of Globoid Cell Leukodystrophy Ilaria Visigalli, Martina Cesani, Margherita Neri, Laura Tononi, Silvia Ungari, Sergio Marchesini, Angela Gritti, Luigi Naldini. THURSDAY, MAY 31 59

61 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Inborn Errors of Metabolism I Storage Diseases Plus Others continued 97 Long-Term Somatic Correction in MPS II Mice Using a Self- Complimentary AAV2 Vector Joseph Muenzer, Lu Kang, Haiyan Fu, Douglas M. McCarty. 98 Active Enzyme Fusions for Therapeutic Targeting of the Central Nervous System Via the Peripheral Circulation in Mucopolysaccharidosis Type IIIB Rafi Awedikian, Elizabeth M. Snella, Brian Spencer, Inder M. Verma, N. Matthew Ellinwood. 99 Effects of LDL Receptor Gene Transfer on Tissue Cholesterol Levels in LDL Receptor Deficient Mice Frank Jacobs, Eline Van Craeyveld, Jan Snoeys, Yingmei Feng, Désiré Collen, Bart De Geest. 100 Detection of CFTR Activity in Individual Epithelial Cells Following Adenovirus-Mediated Delivery of a cdna Encoding Yellow Fluorescent Protein Howard H. Lou, Jamie D. Schwartz, Neil R. Hackett, Ben-Gary Harvey, Philip L. Leopold, Ronald G. Crystal. 101 Long-Term Correction of Hyperphenylalanainemia in a Mouse Model of Phenylketonuria by Intramuscular Delivery raav Pseudotyped with Serotypes 8 or 1 Alexandre Rebuffat, Zhaobing Ding, Beat Thony. 102 Down-Regulation of Hepatic Stearoyl CoA Desaturase1 Prevents Diet-Induced Obesity in Rats Vadim Kroutov, George Aslanidi, Andres Acosta, Sergei Zolotukhin. 103 Low Doses of Self Complimentary Adeno-Associated Virus Vectors Pseudotyped with Nonhuman Primate Serotypes 8 and rh.10 Mediate Highly Efficient Transduction of the Liver Melanie Cochrane, Jenny H. McIntosh, Andrew M. Davidoff, Amit C. Nathwani. 104 AAV-Mediated Gene Transfer to Muscle and Liver of MPS VI Animal Models Alessandra Tessitore, Armida Faella, Thomas O Malley, Monica Doria, Mark Haskins, Alberto Auricchio. 105 Lentivirus-Mediated Gimap5 Gene Delivery To Treat Diabetic BB Rats William R. Osborne, Lanaya Waldron, Margaret Brzezinski, Jessica Fuller, Terry Tupling, Elizabeth A. Rutledge, Ake Lernmark. 106 Treatment of Diabetic Rats with Encapsulated Islets Ofer Yanay, Ian R. Sweet, Lanaya Waldron, Margaret Brzezinski, Merle L. Gilbert, Jessica M. Fuller, Terry Tupling, Ake Lernmark, William R. Osborne. 107 Considerations for AAV8-Mediated Gene Therapy of Pompe Disease Scott D. Bercury, Robin J. Ziegler, Yunxiang Zhu, Joe W. Foley, Nathan Gumlaw, Dwight D. Koeberl, Ronald K. Scheule, Seng H. Cheng. 108 Long-Term Efficacy of a Self-Complementary Adeno- Associated Virus Vector for Phenylketonuria Gene Therapy Akihiro Kume, Takasahi Matsushita, Hiroaki Mizukami, Masashi Urabe, Takashi Okada, Keiya Ozawa. 109 Sleeping Beauty Transposon-Mediated Long-Term α-l- Iduronidase Expression and Correction of Lysosomal Pathology in NOD/SCID Mice with MPS I Elena L. Aronovich, Jason B. Bell, Lalitha R. Belur, Roland Gunther, Brenda Koniar, Patricia A. Schachern, Ilze Matise, Walter C. Low, Chester B. Whitley, R. Scott McIvor, Perry B. Hackett. 110 Recombinant Adeno-Associated Virus Mediated Acyl coa Dehydrogenase (ACD) Gene Replacement in ACD-Deficient Mouse Models Stuart G. Beattie, Qiushi Tang, Eric Goetzmann, Martha Campbell Thompson, Travis Cossette, Thomas Conlon, Dietrich Matern, Gerard Vockley, Terence R. Flotte. 111 AAV1 Mediated Gene Therapy of Neonatal Fabry Mice Koichi Ogawa, Yukihiko Hirai, Masamichi Ishizaki, Yoshitaka Fukunaga, Takashi Shimada. Cardiovascular Gene Therapy I 112 Novel Strategies To Inhibit Ocular Neovascularization Based on Sonic Hedgehog Pathway Blockade Gabriella Cotugno, Enrico M. Surace, Pasqualina Colella, Alberto Auricchio. 113 Transduction of the Vasculature Using raav6 Akshay Krishnamurty, Brian Schultz, James M. Allen, Eric Finn, Caitlin Doremus, Luke M. Judge, Morayma Reyes, Jeffrey S. Chamberlain. 60

62 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Cardiovascular Gene Therapy I continued 114 AAV-Mediated Delivery of Paraoxonase 1 (PON1) to the ApoE-/- Mouse as Gene Therapy for Atherosclerosis Aideen O Doherty, Mira Rosenblat, Michael Aviram, Timothy O Brien. 115 Adeno-Associated Virus 2-Based Gene Delivery of the Angiotensin II Type 2 Receptor Reduces Atherosclerosis and Associated Markers in LDLR Knockout Mice Changping Hu, Abhijit Dandapat, Paul L. Hermonat, Yong Liu, Jawahar L. Mehta. 116 Gene Transfer in Mice Established That Residues Leu261, Trp264 and Phe265 Account for Apolipoprotein E-Induced Dyslipidemia and Affect the Formation of apoe Containing High Density Lipoprotein Konstantinos Drosatos, Kyriakos E. Kypreos, Vassilis I. Zannis. 117 Balanced Co-Expression of VEGF 164 and PDGF-BB Prevents Aberrant Angiogenesis and Improves Hindlimb Ischemia Andrea Banfi, Georges von Degenfeld, Milton J. Merchant, Donald M. McDonald, Helen M. Blau. 118 A Genetically Modified AAV Vector Shows Promise in Ex Vivo Treatment of Human Saphenous Vein Mani A. Daneshmand, Jeffrey Nienaber, Nestor R. Villamizar, Carmelo A. Milano, Dawn E. Bowles. 119 NOS2 Gene Transfer with a Synthetic Vector Inhibits Neointimal Hyperplasia in a Rabbit Vein Graft Model Qing-Hai Meng, Scott A. Irvine, Jean R. McEwan, Stephen L. Hart. 120 Proper Timing of VEGF Expression Is Required for the Formation of Stable and Mature Blood Vessels Sabrina Tafuro, Eduard Ayuso, Serena Zacchigna, Lorena Zentilin, Franca Dore, Mauro Giacca. 121 A Pressured-Freeze Method To Make Multi-Functional Echogenic Liposomes for Image-Guided and Ultrasound- Controlled Oligonucleotide Delivery Shaoling Huang, Robert C. MacDonald, David D. McPherson. 122 AAV2 Pseudotyped with AAV9 Capsid Is an Efficient Vector for Vascular Gene Delivery Ilia Fishbein, Meizan Lai, Robert J. Levy. 123 Intravascular Delivery of Plasmid DNA/Anti-DNA Antibody Complex Immobilized on Coronary Stent Cunxian Song, Xu Jin, Xigang Leng, Lanxia Lu, Robert J. Levy. 124 Transcriptional Targeting of Gene Expression to the Endothelium Using the Roundabout-4 Receptor Promoter Meredith A. Preuss, Justin A. Barnes, Joel N. Glasgow, Yoshiaki Okada, Maaike Everts, William C. Aird, David T. Curiel. 125 Measurement of Flow-Mediated and Nitric Oxide Synthase- Dependent Vasodilation in Living Rats Using High Resolution Ultrasound Christian Heiss, Richard E. Sievers, Nicolas Amabile, Shobha Natarajan, Yerem Yeghiazarians, Matthew L. Springer. Gene Therapy for Muscular Dystrophies 126 Dp116 Expression in Dystrophin/Utrophin Double Knockout (mdx:utrn -/- ) Mice Restores Muscle Mass and Viability but Does Not Correct Dystrophic Pathology Andrea L. H. Arnett, Luke M. Judge, Jeffrey S. Chamberlain. 127 Functional Capacity of Dystrophins N-Terminal Actin Binding Sequences Glen B. Banks, Paul Gregorevic, James M. Allen, Eric E. Finn, Jeffrey S. Chamberlain. 128 Screening of Antisense Oligonucleotides for Human Dystrophin Exon Skipping in a GFP Reporter Culture System with High Sensitivity and Specificity Ehsan Benrashid, Saafan Malik, Allen Zillmer, Randy J. Thresher, Jeffrey Rosenfeld, Qi Long Lu. 129 A Myoblast Expansion System for Improving the Efficiency of Autologous Stem Cell Therapy To Treat Muscular Dystrophy Sheng Li, Brent Fall, Miki Haraguchi, Jeffrey S. Chamberlain. 130 Independent Canine Models of Duchenne Muscular Dystrophy Due to Intronic Insertions of Repetitive DNA Bruce F. Smith, Joe N. Kornegay, Dongsheng Duan. THURSDAY, MAY 31 61

63 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Gene Therapy for Muscular Dystrophies continued 131 In Utero Delivery of Adeno-Associated Virus Vector-Encoding Human Mini-Dystrophin Leads to Functional Correction in Dystrophin Deficient Mice Anthony Y. Tsai, Sasha Bogdanovich, Christina F. Hughes, Masayuki Endo, Jesse Vrecenak, Jeremy Traas, Philip Zoltick, Tejvir S. Khurana, Tim Brazelton, Alan W. Flake. 132 Gene Therapy for Duchenne Muscular Dystrophy by the Helper-Dependent Adenovirus Vector (HDAdv) Mediated Full-Length Dystrophin Expression Masatoshi Ishizaki, Ryoko Kawano, Yuji Uchida, En Kimura, Makoto Uchino, Yasushi Maeda. 133 Complementary Dystrophin and Utrophin Expression Prevents Severe Dilated Cardiomyopathy in 2-Year-Old Mdx Mice Brian P. Bostick, Yongping Yue, Chun Long, Dongsheng Duan. 134 Functional Analysis of alpha-dystrobrevin-3 in Striated Muscle with raav6 Gene Transfer Guy Odom, Glen Banks, James Allen, Marv Adams, Leonard Meuse, Miki Haraguchi, Stan Froehner, Jeffrey Chamberlain. 135 raav Type 8-Mediated Extensive Therapeutic Gene Delivery into Skeltal Muscle of α-sarcoglycan Deficient Mice Akiyo Nishiyama, Beryl N. Ampong, Jin-Hong Shin, Hiroyuki Nakai, Takashi Okada, Shin ichi Takeda. 136 Viral Based Therapeutic for LGMD Type 2D Deficiencies Demonstrates Long-Term Results in Alpha-Sarcoglycan Deficient Animals Louise R. Rodino-Klapac, Christopher J. Shilling, Zarife Sahenk, K. Reed Clark, Jerry R. Mendell. 137 Global Force Improvement by Systemic AAV Delivery of α- Sarcoglycan Transgene in Deficient Mice Marc Bartoli, Francoise Fougerousse, Jérôme Poupiot, Olivier Danos, Isabelle Richard. 138 Ameliorating Dystrophic Pathology Via AAV-Mediated Gene Delivery of Myostatin Propeptide Chunping Qiao, Jianbin Li, Bing Wang, Juan Li, Xiao Xiao. 139 Isolation, Characterization, and Myogenesis of Satellite Cells Derived from Skeletal Muscle Nicholas Ieronimakis, Gayathri Balasundaram, Jeffrey S. Chamberlain, Morayma Reyes. 140 Gait in BioTO-2 and Bio14.6 Dystrophic Hamsters Thomas G. Hampton, Ivo Amende, Ajit Kale, Scott Mccue, Hemmi N. Bhagavan, Anton H. M. Terpstra, Case VanDongen. 141 GNE Vector Development and Testing for Gene Replacement Treatment of Hereditary Inclusion Body Myopathy Chris Jay, Stephan Hinderlich, Daniel Darvish, Gregory Nemunaitis, John J. Nemunaitis, Phillip B. Maples. 142 Spliceosome-Mediated RNA Trans-Splicing for Muscular Dystrophies Chunping Qiao, Jianbin Li, Bing Wang, Juan Li, Xiao Xiao. 143 Effect of Insulin-Like Growth Factor-1 (IGF-1) on Global Gene Expression Profiles in Murine Skeletal Muscle C. Ramana Bhasker, Shaochun Song, Theodore Friedmann. 144 Safety Studies of the Hydrodynamic Limb Vein Delivery Procedure in Primates Julia O. Hegge, Guofeng Zhang, Karl K. Vigen, Thomas M. Grist, Magdolna G Sebestyen, Christine I. Wooddell, Vladimir Subbotin, P. Filippo Adamo, Weijun Cheng, Thierry Huss, Serge Braun, James E. Hagstrom, Jon A. Wolff. Vaccinating against Pathogens Using Gene Delivery 145 Vaxfectin -Formulated Plasmid DNA Measles Vaccine Elicits Protective Immune Response in Non-Human Primates Adrian Vilalta, Chien-Hsiung Pan, Gretchen Jimenez, Robert Adams, Alain Rolland, David C. Kaslow, Diane E. Griffin. 146 DNA Vaccination in Combination with Constant-Current Electroporation Leads to an Increased Immune Response in Mice, Rabbits and Nonhuman Primates Ruxandra Draghia-Akli, Amir S. Khan, Ranajit Pal, Anthony Cristillo, Deborah Weiss, Lauren Hirao, David B. Weiner. 147 Novel Strategy for Generation of Mucosal Immune Responses Against HIV-1 Following Systemic Vaccination Michele A. Kutzler, Kimberly A. Schoenly, Karuppiah Muthumani, Rose M. Parkinson, Henry Maguire, Kenneth Ugen, David B. Weiner. 62

64 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Vaccinating against Pathogens Using Gene Delivery continued 148 Multivalent Adenovector-Based Malaria Vaccine Induces High Levels of Functional Antibodies Joseph T. Bruder, Ping Chen, Svetlana Konovalova, Keith Limbach, Damodar Ettyreddy, Maureen E. Stefaniak, Noelle B. Patterson, Joseph J. Campo, Sheng Li, C. R. King, Carole A. Long, Denise L. Doolan. 149 Nasal Delivery of Adenovirus-Based Vaccine Bypasses Pre- Existing Immunity to the Vaccine Carrier and Improves the Quality of the Immune Response Maria Croyle, Ami Patel, Yi Zhang, Kaylie Tran, Michael Gray, Jim Strong, Heinz Feldmann, Gary P. Kobinger. 150 Vector Prime-Protein Boost Vaccine for Avian and Human Influenza Viruses Yucheng Tang, Hakan Akbulut, Jonathan Maynard, Line Pedersen, Albert Deisseroth. 151 Novel Genetic Vaccine for Anthrax Based on Recombinant AAV Vectors Te-Hui Liu, Jon Oscherwitz, Bruce Schnepp, Kemp Cease, Philip R. Johnson. 152 Immunogenicity of a Cytokine Enhanced Multi-Antigen pdna Vaccine with and without In Vivo Electroporation in Rhesus Macaques M. A. Egan, A. Luckay, I. Mathiesen, J. H. Eldridge, R. Kjeken. 153 Adenovector-Based Malaria Vaccine Induces Strong T-Cell and Antibody Responses Against Multiple Pre-Erythrocytic- Stage Antigens Joseph T. Bruder, Elena Semenova, Charlie Thomas, Duncan McVey, Svetlana Konovalova, Keith Limbach, Maureen E. Stefaniak, Noelle B. Patterson, Joseph J. Campo, Sheng Li, C. R. King, Denise L. Doolan. 154 Severe Acute Respiratory Syndrome Coronavirus-Like Particles (SARS-CoVLPs) Stimulate Human Monocyte- Derived Dendritic Cell Activity and Induce Antibody Response in Mouse Model Chih-Chun Wen, Pen-Wen Hsiao, Yi-Chun Yeh, Sheng-Chu Kuo, Li-Jiau Huang, Aravindaram Kandan, Ning-Sun Yang. 155 Partial Protection Against H5N1 Influenza in Mice with a Single Dose of a Chimpanzee Adenovirus Vector Expressing Nucleoprotein Soumitra Roy, Gary P. Kobinger, Joanita Figueredo, Roberto Calcedo, Darwyn Kobasa, James M. Wilson. 156 Protective Immune Responses Against Porcine Circovirus Type 2 Infection Generated by a Recombinant Modified Vaccinia Virus Expressing the Viral ORF Antigens as Vaccines Kandan Aravindaram, Tsun Yung Kuo, Chun Wen Lan, Hsiu Hui Yu, Pei Hsueh Wang, Lu Tang Chiu, Yu San Chen, Ning Sun Yang. 157 Enhancement of Antibody and Cellurar Responses to Malaria DNA Vaccines by In Vivo Electroporation Carlota Dobaño, Georg Widera, Dietmar Rabussay, Denise L. Doolan. 158 Adenovirus-Mediated Delivery of a Y. pestis V Antigen Variant with Reduced Immunomodulatory Properties Elicits Comparable Serum Anti-V Antigen IgG Titers to Adenovirus- Mediated Delivery of Wild-Type V Antigen Carolina Sofer-Podesta, Julie L. Boyer, Ronald G. Crystal. 159 Recombinant Adeno-Associated Virus Vectors Effectively Immunize Rabbits Via Intramuscular and Intranasal Routes Clifford J. Beall, Philip R. Johnson. 160 In Vivo Immunogenic Responses by Tuberculosis Vaccination with Sendai-Virosomal pdna Encoding Mycobacterial Antigen 85 Complex A (mag85a) In Ho Song, Yoen Kyung Lee, Jung Seok Kim, Kyung Hye Koo, Sang Nae Cho, Yong Serk Park. 161 In Vivo Electroporation Promotes Intra-Muscular DNA Uptake, Protein Expression, and Inflammation at the Site of Injection, Which Greatly Enhances the Immunogenicity of Hepatitis C Virus Non-Structural 3/4A DNA Gustaf N. Ahlen, Jonas Soderholm, Torunn Tjelle, Rune Kjeken, Iacob Mathiesen, Matti Sallberg. 162 Antigenic Correlates of Immunity to Avian Influenza Provided by Consensus DNA Immunogens Dominick J. Laddy, Jian Yan, Darwyn Kobasa, Gary P. Kobinger, David B. Weiner. Cancer Immunotherapy I 163 Adenoviral Vector Prime-Protein Boost Vaccine for Melanoma Yucheng Tang, Jonathan Maynard, Albert Deisseroth. THURSDAY, MAY 31 63

65 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Cancer Immunotherapy I continued 164 Enhanced Anti-Tumor Effect of Modified Vaccinia Virus Expressing Human-gp100 Antigen and Rantes in Cancer Vaccine Approach Kandan Aravindaram, Hsiu Hui Yu, Chun Wen Lan, Ning Sun Yang. 165 Heat Shock Protein 70-Antigen Fusion Protein Expressing DNA Vaccine for Cancer Immunotherapy Ayumi Yamaoka, Seiji Takemoto, Makiya Nishikawa, Tomoya Yata, Yuji Ohno, Yoshinobu Takakura. 166 The Opposite Effect of Lipopolysaccharide on the Antitumor Therapeutic Efficacy of DNA Vaccine Chi-Chen Lin, Meng-Chi Yen, Ming-Derg Lai. 167 Macrophages Transduced with an Adenoviral Vector Expressing Glipr1 Suppress Tumor Growth and Metastasis in a Preclinical Metastatic Prostate Cancer Model Ken-ichi Tabata, Masami Watanabe, Kohei Edamura, Guang Yang, Jianxiang Wang, El Moataz Abdel Fattah, Dov Kadmon, Timothy C. Thompson. 168 Allograft Vaccine for Epithelial Cancers Yucheng Tang, Hakan Akubulut, Jonathan Maynard, Line Pedersen, Albert Deisseroth. 169 Combination Oncolytic Herpes Simplex Virus and Dendritic Cell Immunotherapy for the Treatment of Established Murine Neuroblastomas Christopher J. Farrell, Cecile M. Zaupa, Robert L. Martuza, Samuel D. Rabkin, William T. Curry. 170 Treatment of Metastatic Renal Cell Cancer Using Multiple IV Administrations of IL-12 Plasmid Nanopolyplexes Jason G. Fewell, Jennifer S. Rice, Miranda Hickman, Majed M. Matar, Elaine Brunhoeber, Casey Pence, Gregory Slobodkin, Danny H. Lewis, Khursheed Anwer. 171 Preclinical Evaluation of an icd40-dendritic Cell Vaccine for Prostate Cancer Natalia Lapteva, Mamatha R. Seethammagari, Brent A. Hanks, Kevin M. Slawin, David M. Spencer. 172 Long Term Follow up of GVAX in NSCLC (MCCRC Results) John J. Nemunaitis, Phillip B. Maples, Neil Senzer, Rosemarie Arzaga, Kristen Hege. 173 Her-2/neu DNA Immunization through Constant-Current Electroporation Provides Long-Term Protection from Autochthonous Mammary Carcinomas Amir S. Khan, Claudia Curcio, Elena Quaglino, Federica Cavallo, Guido Forni, Ruxandra Draghia-Akli. 174 B Cells Can Function as Tumor Antigen Presenting Cells In Vivo James F. Curtin, Tamer Fakhouri, Chunyan Liu, Pedro R. Lowenstein, Maria G. Castro. 175 Single Dosage Surface Functionalized Microparticles for Combinatorial DNA and Oligonucleotide/Adjuvant Immunization Ankur Singh, Bilal Ghosn, Kassandra Thomson, Krishnendu Roy. 176 Necrotic Tumor Cell Extracts Stimulate TLR2-Dependent T Cell Mediated Tumor Regression James F. Curtin, Gwendalyn D. King, Kathrin S. Michelsen, Carlos Barcia, Tamer Fakhouri, Daniel Larocque, Marianela Candolfi, Begonya Comix-Anduix, Chunyan Liu, Kurt M. Kroeger, Daniel Resnick, Moshe Arditi, Antoni Ribas, Pedro R. Lowenstein, Maria G. Castro. 177 In Vivo Phagocytic Activity of Antigen Presenting Cells Infiltrating Intracranial Glioblastomas in Rats Treated with Adenoviruses Expressing TK and Flt3L Marianela Candolfi, A. K. M. G. Muhammad, James F. Curtin, Chunyan Liu, Kurt M. Kroeger, Pedro R. Lowenstein, Maria G. Castro. 178 Spontaneous Glioblastoma in Dogs: Preclinical Model for High Capacity Adenoviral Vector-Mediated Experimental Gene Therapy M. Candolfi, J. Curtin, W. S. Nichols, A. K. M. G. Muhammad, M. Puntel, G. D. King, W. Xiong, K. M. Kroeger, C. Liu, G. E. Pluhar, E. A. Mc Niel, J. R. Ohlfest, A. B. Freese, P. F. Moore, D. Palmer, P. Ng, J. D. Young, P. R. Lowenstein, M. G. Castro. 179 A Phase I Clinical Trial of Liposome-Mediated Interferonbeta Gene Therapy for High-Grade Gliomas Toshihiko Wakabayashi, Masaaki Mizuno, Atsushi Natsume, Masazumi Fujii, Jun 64

66 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Cancer Immunotherapy I continued 180 Induction of Anti-Tumor Immunity by the Baculovirus Autographa Californica Multiple Nuclear Polyhedrosis Virus Masayuki Kitajima, Takayuki Abe, Naoko Miyano-Kurosaki, Toshinori Nakayama, Hiroshi Takaku. 181 Eradication of Multifocal Glioma in a Syngeneic Glioblastoma Multiforme with Ad-Flt3L and Ad-HSV1-TK Gwendalyn D. King, A. K. M. Ghulam Muhammad, James F. Curtin, Carlos Barcia, Mariana Puntel, Chunyan Liu, Sarah B. Honig, Marianela Candolfi, Sonali Mondkar, Pedro R. Lowenstein, Maria G. Castro. 182 Progress in the Clinical Development of a Novel IL-12 Gene Therapeutic for the Treatment of Recurrent Ovarian Cancer Khursheed N. Anwer, Mack N. Barnes, Mathew L. Anderson, Connie McCombs, Souheil Saddekni, Sharmila Makhija, Ivan Maya, Jason G. Fewell, Margit M. Janat-Amsbury, Danny H. Lewis, Ronald D. Alvarez. 183 Interferon-alpha Gene Transfer Enhances Antitumor Activity of Allogeneic Hematopoietic Stem Cell Transplantation Against Solid Cancers Kazunori Aoki, Hidehiko Hara, Akihiko Kobayashi, Kimiko Yoshida, Teruhiko Yoshida. 184 Induction of Efficient Antitumor Immunity to Murine Neuroblastoma Using Dendritic Cells Highly Activated by Temperature-Sensitive Mutant SeV/dF: A Preclinical Efficacy Study Sakura Tanaka, Yoshikazu Yonemitsu, Kyosuke Tatsuta, Sinji Okano, Haruhiko Kondo, Satoko Shibata, Tatsuro Tajiri, Sachiyo Suita, Tomoaki Taguchi, Yasuji Ueda, Makoto Inoue, Mamoru Hasegawa, Katsuo Sueishi. 185 Development of Immunotherapeutics for Mantle Cell Lymphoma Linhong Li, Joshua Brody, Stephanie Felller, Cornell Allen, Rama Shivakumar, Madhusudan V. Peshwa, Joseph C. Fratantoni, Ron Levy, Linda N. Liu. 186 A Phase I Study of Antitumor Vaccination Using Lung Cancer Cells Genetically-Modified To Express Alpha(1,3)galactosy ltransferase (αgt) in Patients with Stage IV, Refractory or Recurrent Non-Small Cell Lung Cancer (NSCLC) John C. Morris, John E. Janik, Nicholas Vahanian, Nancy Harold, Deirdre O Mahony, Kamal Sharma, Gabriela R. Rossi, Henry Y. Lejukole, Lucinda Tennant, Stefania Pittaluga, Jason C. Steel, Charles J. Link. 187 Adenovirus Expressing Interleukin 6 Increases the Anti- Tumor Activity of Dendritic Cells Sung-Min Oh, Hwang-Jae Lee, Jae-Gyun Jeong, Jin-A Chae, Seok Kang, Hae-Sook Ahn, Soo-Min Choi, Seong-Hyun Ho, Hong-Chan Cho, Jong- Mook Kim, Su-Jeong Kim. 188 Phase II Study of Intratumoral (IT) OncoVexGM-CSF, an Immune-Enhanced Oncolytic Herpes Simplex-1 Virus (HSV- 1), in Patients with Stage IIIc/IV Malignant Melanoma (MM): A Preliminary Report of a Complete Response of Target and Reporter Lesions Neil Senzer, Michael Nemunaitis, John J. Nemunaitis, Minal Barve. 189 Antitumoral Efficacy of Semliki Forest Virus Vectors Expressing IL-12 in Liver Tumor Models Juan R. Rodriguez-Madoz, Maider Zabala, Erkuden Casales, Maite Alfaro, Jesus Prieto, Gabriela Kramer, Cristian Smerdou. 190 Induction and Enhancement of Cancer Immunity by Ion Particle Beam and Dendritic Cell Treatment of Murine Squamous Cell Carcinoma Yasuji Ueda, Akinao Matsunaga, Kumi Ishida, Yasuo Yoneyama, Shigeru Yamada, Tomonori Kato, Yui Harada, Hiroaki Kinoh, Makoto Inoue, Mamoru Hasegawa, Hirohiko Tsujii, Takenori Ochiai, Yoshikazu Yonemitsu. 191 TGF-Beta Inhibition Augments Immuno-Gene Cytokine and Vaccine Therapy Samuel Kim, George Buchlis, Veena Kapoor, Jing Sun, Larry Kaiser, Leona Ling, Steven Albelda. Cancer: Apoptosis & Suicide 192 Anti-Tumor Activity of Mesenchymal Stem Cells Producing IFN-α in a Mouse Melanoma Lung Metastasis Model Changchun Ren, Sanjay Kumar, Diptiman Chanda, Selvarangan Ponnazhagan. 193 Novel Adenoviral Vector Two-Step Transcription Amplification Expression System (TSTA) for Expressing CDK2-Associated Cell Cycle Regulators for Cancer Gene Therapy Marxa L. Figueiredo, May N. Chen, Makoto Sato, Russell Powell, Ewa Jaruga, Walter Rayford, Lily Wu. 194 Induction of Apoptosis in a Human Testicular Cancer Cell Line by Overexpression of REIC/Dkk-3 In Vitro and In Vivo Ryuta Tanimoto, Abarzua Fernando, Masakiyo Sakaguchi, Yasutomo Nasu, Hiromi Kumon, Nam-ho Huh. THURSDAY, MAY 31 65

67 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Cancer: Apoptosis & Suicide continued 195 Conditionally-Replicative Adenovaral Vectors with Bioluminescence-Based Replication Monitoring Capability for Lung Cancer Julia Davydova, Eric J. Brown, Masato Yamamoto. 196 Adenoviral Delivery of Secretable Trimeric TRAIL in Combination with Conventional Chemotherapy for Brain Tumors Induces Synergistic Tumor Suppression and Improves Survival in an Intracranial Human Malignant Glioma Xenograft Mouse Model Moonsup Jeong, Yong-Sam Kwon, Soon-Hye Park, Min-Tae Park, Chae- Young Kim, Kang-Won Son, Yong Ko, Paul D. Robbins, Dai-Wu Seol, Byong-Moon Kim. 197 mda-7 (INGN-241) Kills Chemoresistant Cancer Cells and Restores Chemosensitivity to Cisplatin Began Gopalan, Carlos Rached, Daniel Pearson, Manish Shanker, Sunil Chada, Rajagopal Ramesh. 198 Engineering Cytosine Deaminase/Uracil Phosphoribosyltransferase Fusion for Improved Cancer Gene Therapy Andressa Ardiani, Michi Fuchita, Margaret E. Black. 199 Overexpression of Mitochondrial Localized (mt)-catalase Further Increases Radioresistance Induced by Manganese Superoxide Dismutase (MnSOD) Overexpression in 32D cl 3 Murine Hematopoietic Progenitor Cells Michael W. Epperly, J. Andres Melendez, Xichen Zhang, Darcy Franciola, Tracy Smith, Joel S. Greenberger. 200 Identification of Genetics Alterations That Confer Resistance to Cisplatin in Ovarian Cancer Ying Liu, Pavel Sova, Robert Strauss, Zongyi Li, Nicole Urban, Charles Drescher, Andre Lieber. 201 Replication Deficient Adenovirus Carrying Apolipoprotein (a) Kringles Suppress Tumor Growth in NSCLC Xenograft Tumor Model Kyuhyun Lee, Sung-Tae Yun, Hong-Kyu Lee, Eui-Cheol Jo. 202 Combined Effects of Ad5F35-TK/GCV Gene Therapy and Gemcitabine in Bladder Transitional Cell Carcinoma Weiguo Jian, Guru Sonpavde, Yamashita Hideyuki, Hao Liu, Seth P. Lerner. 203 Systemic Stable Radioprotection by Intravenous Administration of Manganese Superoxide Dismutase- Plasmid Liposomes Joel S. Greenberger, Tracy Smith, James J. Schlesselman, Michael W. Epperly. 204 Gene Therapy of Pancreatic Carcinoma Based on Gemcitabine Chemosensitization Using Phosphorylating Fusion Gene Transfer Fabienne Vernejoul, Barbara Bournet, Anny Souque, Hubert Lulka, Gilles Cambois, D. Drocourt, Lucien Pradayrol, Gérard Tiraby, Pierre Cordelier, Louis Buscail. 205 Knockdown of Lifeguard (LFG/FAIM2) in Glioblastoma Cells Increases Sensitivity to FasL and Doxorubicin Bryan C. Nikolai, Nikunj V. Somia. 206 Systemic Osteoprotegerin Gene Therapy Restores Tumor- Induced Bone Loss in a Therapeutic Model of Breast Cancer Bone Metastasis Diptiman Chanda, Tatyana Isayeva, Sanjay Kumar, April F. Szafran, Kurt R. Zinn, Selvarangan Ponnazhagan. 207 Cell-Death Induction of Primary Melanocytes Cultures after Transfection of p(hsv-tk) by a Synthetic Reagent (KLN-47) and Followed by Addition of GCV Tristan Montier, Delphine Le Goupil, Ulysse Pereira, Pascal Delépine, Tony Le Gall, Pierre Lehn, Laurent Misery, Claude Férec. 208 Inducing Autophagy with a Gene Therapy Combined System Provides Successful Treatment Against Cancer Ricardo Gargini, Vega Garcia-Escudero, Marta Izquierdo. 209 Connexin 43 Expression in Glioblastoma for Suicide Gene Therapy Sylvine Cottin, Leo Cantin, Peter V. Gould, Manuel Caruso. 210 Adenovirus-Mediated Thymidine Kinase Gene Transfer into Uterine Artery of Pregnant Rabbits Combined with Gansiclovir Treatment Does Not Affect the Fertility of the Does Anniina Laurema, Mervi Riekkinen, Tommi Heikura, Elisa Vahakangas, Hannu Manninen, Seppo Heinonen, Seppo Yla-Herttuala. 211 Restoration of Mutant p53 Function through the Alteration of Pre-mRNA Splicing Pattern Daniel J. Catron, Paloma H. Giangrande, Kristi D. Viles, Bruce A. Sullenger. 66

68 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Cancer: Apoptosis & Suicide continued 212 Systemic Expression of Pro-Opiomelanocortin Prohormone Induces Melanogenesis and Tumor Regression in B16-F10 Melanoma Guei-Sheung Liu, Ming-Hong Tai. 213 NK4, Competitive Antagonist for HGF, Therapy Against Brain Tumors by Using with Replication-Incompetent HSV-1 Vector: 1764/4-/NK4 Yoko Matsushita, Naosuke Nonoguchi, Kunio Matsumoto, Toshikazu Nakamura, Robert S. Coffin, Shin-ichi Miyatake, Toshihiko Kuroiwa. Cancer: Targeted Therapy I 214 Genetic Engineering of a Human CD20-Specific Chimeric T-Cell Receptor and Co-Expression of a Linked Inducible Caspase-9 Suicide Gene Yukang Lin, Jinjuan Wang, Carolina Berger, Stanley Riddell, David M. Spencer, Oliver W. Press. 215 Soluble Bi-Specific Adapter-Mediated Targeting of Herpes Simplex Virus Type 1 (HSV-1) to the Carcinoembryonic Antigen (CEA) Hyunjung Baek, Namchul Cho, Eugene Jeon, Kyuseon Ahn, Yoohun Noh, Kee-ho Lee, Masahide Kuroki, William F. Goins, Justus B. Cohen, Joseph C. Glorioso, Heechung Kwon. 216 Oncolytic Adenoviruses Kill Breast Cancer Stem Cells Minna Eriksson, Gerd Bauerschmitz, Pekka Virkkunen, Maija Tarkkanen, Kilian Guse, Tanja Hakkarainen, Anna Kanerva, Akseli Hemminki. 217 Systemic Administration of a Replicating Adenovirus, Transcriptionally Targeted to Angiogenesis, Reduced Lung Metastases Burden in Cotton Rats Michael Peled, Aviv Shaish, Shoshana Greenberger, Avi Katav, Reshef Tal, Eyal Breitbart, Dror Harats. 218 Preclinical Studies on Matrix Metalloprotease-Activatable Measles Viruses for the Virotherapy of Solid Liver Tumours Christian J. Buchholz, Michael D. Muehlebach, Thomas Schaser, Martina Zimmermann, Sorin Armeanu, Michael Bitzer, Ulrich M. Lauer, Roberto Cattaneo, Klaus Cichutek. 219 Analysis of Mechanisms of Synergy between Irradiation and Oncolytic Adenoviruses in the Treatment of Hormone Refractory Prostate Cancer M. Rajecki, M. Tenhunen, T. Hakkarainen, S. Hautaniemi, O. Monni, U.-H. Stenman, K. Guse, R. M. Desmond, D.-T. Chen, A. Kanerva, A. Hemminki. 220 Sleeping Beauty-Mediated Antiangiogenic Gene Therapy for Metastatic Colorectal Cancer Lalitha Belur, Kelly Podetz-Petersen, Brent Sorenson, S. Ramakrishnan, Daniel Saltzman, Perry B. Hackett, R. Scott McIvor. 221 Directed Evolution Generates a Novel Potent and Selective Ad3/Ad11p Chimeric Oncolytic Virus for the Treatment of Colon Cancer Terry W. Hermiston, Irene Kuhn, Paul Harden, Maxine Bauzon, Shaoheng Ni, Andre Lieber, Kerry Fisher, Len Seymour, Tony Reid, Steve Thorne, Gabor Rubanyi, Richard Harkins. 222 MicroRNA Signature of Human Prostate Cancer and Its Applicability in Identifying Early Relapse Disease Chris Jay, John J. Nemunaitis, Pat Fulgham, Patrick Chen, Aron Eklund, Neil Senzer, Jian Han, Alex W. Tong. 223 VEGF-Targeted Artificial Zinc Finger Protein-Expressing Adenovirus Downregulates Angiogenesis and Leads to Enhanced Anti-Tumor Effects Sung-Mi Jung, Yoona Kang, Byoung Chul Cho, Ji-Hoon Ryu, Min-Ju Kim, Joo-Hang Kim, Chae-Ok Yun. 224 Systemic Administration of a Dual-Targeted Lentiviral Vector Leads to Specific Transduction of Prostate Cancer Bone Metastases In Vivo Nonia Pariente, Kouki Morizono, Mandeep Virk, Frank Petrigliano, Robert E. Reiter, Jay R. Lieberman, Irvin S. Y. Chen. 225 A Targeted, Adenovirus-Mediated Histone Deacetylase (HDAC) Specific shrna Approach Enhanced Cancer Radiation Therapy Shanling Liu, He Wang, Zhonghui Yang, Jiangao Zhu, Fang Li, Takashi Kon, Chuan-Yuan Li. 226 PTTG1 Is a Prognostic Factor and a Potential Therapeutic Target in Esophageal Squamous Cell Carcinoma Tetsuo Ito, Yutaka Shimada, Takatsugu Kan, Stefan David, John M. Abraham, Yuriko Mori, Yulan Cheng, Bogdan Paun, Zhe Jin, Alexandru Olaru, Rachana Agarwal, James P. Hamilton, Jian Yang, Stephen J. Meltzer, Fumiaki Sato. THURSDAY, MAY 31 67

69 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Cancer: Targeted Therapy I continued 227 Ex Vivo Virus Production in Fresh Tissue Discs Predicts In Vivo Tumor Response in Xenograft Cancer Models Directly Injected with an Oncolytic HSV Mutant Mark A. Currier, Rebecca A. Gillespie, Timothy P. Cripe. 228 Relaxin Expression from Tumor-Targeting Adenoviruses and Its Intratumoral Spread, Apoptosis Induction, and Efficacy In-Wook Kim, A-rum Yoon, Jing-Hua Huang, Young-Sook Lee, Jung- Sun Lee, Joo-Hang Kim, Chae-Ok Yun. 229 Identification of New Therapeutic Targets To Potentiate Oncolytic Adenovirus Yaohe Wang, Xingbo Zhao, Ruthi Gangeswaran, Vipul Bhakta, Jennelle Francis, Yunhan Zhang, Weidong Zang, Ziming Dong, Nick Lemoine. 230 Prevention of Kupffer Cell Viral Clearance of Genetically and Non Genetically RGD Targeted Adenovirus, upon Systemic Administration, by the Use of Polinosinic Acidic Hidde J. Haisma, Jan A. A. M. Kamps, Gera K. Kamps, Anna Rita Bellu. 231 Development of Antineovascular RNAi Cancer Therapy Targeting to Argonaute2 Tomohiro Asai, Yuko Suzuki, Saori Matsushita, Junichi Yokota, Tatsuhiro Ishida, Hiroshi Kiwada, Naoto Oku. 232 Potential Significance of OV Induced Cyr61 as a Biomarker for OV, and Its Impact on Therapy Kazuhiko Kurozumi, Jayson Hardcastle, Roopa Thakur, Joshua Shroll, Akihiro Otsuki, E. Antonio Chiocca, Balveen Kaur. 233 Non-Invasive Bioluminescence and PET Imaging Detection of Prostate Cancer Lymph Node Metastases by Transcriptionally-Targeted Adenoviral Vectors Jeremy Burton, Mai Johnson, Shuwen Ko, Makoto Sato, Lily Wu. 234 Inhibition of Melanoma Development by Single Dose Administration of htertc27 Viral Cocktail in C57BL/6 Mice Xicai Wang, Longfei Huo, Hong Yao, Yan-Ting Qi, Hsiang-fu Kung, Marie C. Lin. 235 Chemoprotection and Persistent Dihydrofolate Reductase Transgene Expression in Primary and Secondary Transplant Recipients of Lentivirally-Transduced Marrow Jennifer L. Gori, Kelly Podetz-Pedersen, Debra Swanson, Andrea D. Karlen, Nikunj V. Somia, R. Scott McIvor. 236 An Improved Human Carboxylesterase for Use in Enzyme/ Prodrug Therapy Approaches with CPT-11 Monika Wierdl, Lyudmila Tsurkan, Carol C. Edwards, Matthew R. Redinbo, Randy M. Wadkins, Philip M. Potter. 237 Efficient, Specific Propagation of a Transcriptionally Targeted Oncolytic Adenovirus To Treat Prostate Cancer Makoto Sato, Russell Powell, Michael Carey, Sanjiv S. Gambhir, Lily Wu. Hematologic I 238 Vector Integration Analysis in Mature T Cells Following PBL Gene Therapy in ADA-SCID Reveals a Cell-Type Specific Insertion Profile Luca Biasco, Barbara Cassani, Massimiliano Mirolo, Grazia Andolfi, Immacolata Brigida, Alessandra Recchia, Fulvio Mavilio, Maria Grazia Roncarolo, Claudio Bordignon, Alessandro Aiuti. 239 Insertional Mutagenesis of Retroviral Vectors Is Dependent on Transgenes in a Canine Stem Cell Gene Therapy Model Xiao-Bing Zhang, Brian C. Beard, Grant D. Trobridge, R. Keith Humphries, Hans-Peter Kiem. 240 In Vivo Expansion of Integration Clones Caused by Retroviral Activation of MDS1-EVI1, PRDM16 or SETBP1 in a Successful Clinical Chronic Granulomatous Disease (CGD) Gene Therapy Trial Kerstin Schwarzwaelder, Manfred Schmidt, Annette Deichmann, Marion G. Ott, Stefan Stein, Hanno Glimm, Ulrich Siler, Dieter Hoelzer, Reinhard Seger, Manuel Grez, Christof von Kalle. 241 Stable Reduction of CCR5 by RNA Interference through Hematopoietic Stem Cell Transplant in Non-Human Primates Dong Sung An, Robert E. Donahue, Masakazu Kamata, Betty Poon, Mark Metzger, Mao Si-Hua, Aylin Bonifacino, Allen E. Krouse, Jean-LUC Darlix, David Baltimore, F. Xiao-Feng Qin, Irvin S. Y. Chen. 242 Adenovirus-Mediated Systemic Overexpression of Sonic Hedgehog Results in Expansion of Diaphyseal Trabecular Bone with Associated Increase in the Hematopoietic Stem Cell Niche Maija Kiuru, Chisa Hidaka, Ralf-Harto Hubner, Anja Krause, Philip L. Leopold, Ronald G. Crystal. 68

70 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Hematologic I continued 243 Insertion Site Analysis Following Gammaretroviral Gene Therapy for Canine Leukocyte Adhesion Deficiency Reveals Lack of Genotoxicity Mehreen Hai, Thomas R. Bauer, Jr, Laura M. Tuschong, Yuchen Gu, Xiaolin Wu, Dennis D. Hickstein. 244 Role of Chromatin Structure in Integration Site Selection of Helper-Dependent Ad5/35 Vectors Carrying the Globin LCR Hongjie Wang, Pavel Sova, Karol Bomstyk, Qiliang Li, George Stamatoyannopolous, Andre Lieber. 245 Hematopoietic Stem Cell Gene Delivery: High Efficiency Gene Transfer to HSC In Vivo with Long-Term Expression of Anti- HIV Transgenes after Intramarrow Injection SV40-Derived Vectors Alena A. Chekmasova, Mahender Singh, Jean-Pierre Louboutin, David S. Strayer. 246 Very High Levels of Expression of the O 6 Methylguanine- DNA-Methyltransferase P140K Mutant Results in an In Vivo Engraftment Defect Michael D. Milsom, Chad E. Harris, Axel Schambach, Jeff Bailey, Emily Broun, Christopher Baum, Geoffrey P. Margison, Ina Rattmann, Moran Jerabek-Willemsen, Thomas Moritz, Jurgen Thomale, Elke Grassman, David A. Williams. 247 Long-Term Efficacy and Safety of Hematopoietic Stem Cell Gene Therapy in the Murine Model of Wiskott-Aldrich Syndrome Francesco Marangoni, Marita Bosticardo, Samantha Scaramuzza, Cristina Panaroni, Sara Trifari, Michela Locci, Elena Draghici, Anne Galy, Luigi Naldini, Alessandro Aiuti, Loïc Dupré, Anna Villa, Maria- Grazia Roncarolo. 248 Evidence of Globin Lentiviral Vector-Induced Genotoxicity in a Murine Model of Sickle Cell Disease Pestina I. Pestina, Hargrove W. Hargrove, Huifen Zhao, Kumar Srivastava, Gray T. John, Derek A. Persons. 249 Gene Therapy in Adenosine Deaminase-Deficient Children with Autologous Bone Marrow Cells Transduced with Two Retroviral Vectors after Withdrawal of Enzyme Replacement Treatment and Low-Dose Chemotherapy Robert A. Sokolic, Greg Podsakoff, Linda Muul, Barbera Engel, Elizabeth K. Garabedian, Denise Carbonaro, Joanna Ireland, Laura M. Tuschong, Michael S. Hershfield, Donald B. Kohn, Fabio Candotti. 250 Lentiviral-Transduced HSC Lead to Long-Term Correction of ß-Thalassemia by In Vivo Selection of Erythroid Progenitors Annarita Miccio, Rossano Cesari, Claudia Rossi, Francesca Tiboni, Francesca Sanvito, Maurilio Ponzoni, Samantha Routledge, Michael Antoniou, Giuliana Ferrari. 251 Development of Leukemia/Lymphoma after the Long Latency Period in an X-SCID Mouse Model Treated by Retroviral Gene Therapy Hideki Hanawa, Takashi Shimada. Immune Responses to Transgene Products 252 Immune Responses Against Tetracycline-Dependent Transactivators Do Not Affect Long-Term Transgene Expression Delivered by a Helper-Dependent Adenoviral Vector (HC-Ad) Weidong Xiong, Mariana Puntel, Sonali Mondkar, James F. Curtin, Marianela Candolfi, Kurt M. Kroeger, Chunyan Liu, Daniel Larocque, Donna Palmer, Philip Ng, Pedro R. Lowenstein, Maria G. Castro. 253 Regulation of Factor VIII-Specific Immune Responses in a Plasmid DNA Treated Hemophilia A Mouse Model by Transgenic FOXP3 + CD4 + Regulatory T Cells Benjamin R. Harmeling, Steven Ziegler, Troy Torgerson, Liping Chen, Baowei Peng, Hans D. Ochs, David J. Rawlings, Carol H. Miao. 254 AAV In Vivo Gene Transfer Suppresses Adaptive Immune Responses Against α-1 Antitrypsin Ekaterina Breous, Suryanarayan Somanthan, Luk H. Vandenberghe, James M. Wilson. 255 Cross Presentation of AAV2 Capsids Activates Cytotoxic T Cells and Hinders Muscle-Directed Gene Transfer but Does Not Render Hepatocytes Effective Cytolytic Targets Lili Wang, Joanita Figueredo, Roberto Calcedo, Jianping Lin, James M. Wilson. 256 Ultrasound Guided Gene Transfer to the Fetal Thymus Results in Thymic Epithelial Cell Transduction and Deletion of Transgene Specific T Cells Masayuki Endo, William H. Peranteau, Phillip W. Zoltick, Antoneta Radu, Nidal Muvarak, Alan W. Flake. THURSDAY, MAY 31 69

71 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Immune Responses to Transgene Products continued 257 Characteristic of Responsive CD8+ T Cells to AAV 2/8 and Ad C7 HIV Gag Vector Vaccines Jianping Lin, Joanita M. Figueredo, Yan Zhi, Roberto Calcedo, Lili Wang, Zhe Zhang, James M. Wilson. 258 Lentiviral-Mediated Gene Transfer to Lung Activates Transgene and Vector Specific T Cells Maria L. Limberis, Christie Bell, Roberto Calcedo, Luk H. Vandenberghe, Deirdre McMenamin, Regina Munden, Di Wu, Julie C. Johnston, Peter Bell, James M. Wilson. 259 Characterization of AAV Capsid Responses in Humans Using Normal Donor Splenocytes Daniel J. Hui, Federico Mingozzi, Etiena Basner-Tschkarajan, Shyrie Edmonson, Katherine A. High. 260 Tolerance Induction to FIX in Mice with Pre-Existing Anti-FIX Immunity Following Direct Intramuscular Injection of AAV Ellen F. Cohn, Meagan E. Kelly, Jiacai Zhuo, Hengjun Chao. 261 Antigen-Specific Tolerance Induction by Transcriptional Targeting of Dendritic Cells with a Novel Lentiviral Vector Christiane Dresch, Thomas Brocker. 262 Suppression of Hyperglycemia in Nod Mice after Inoculation with Recombinant Vaccinia Viruses Bela Denes, Jie Yu, Nadja Fodor, Zsuzsanna Takatsy, Istvan Fodor, William H. R. Langridge. 263 Efficient Induction of Immune Tolerance to FIX Following Direct Intramuscular Gene Transfer Ellen F. Cohn, Meagan E. Kelly, Jiacai Zhuo, Hengjun Chao. 264 Induction of FIX Tolerance by Direct Intramuscular Injection of AAV Is AAV-Dose/FIX-Level Dependent Ellen F. Cohn, Meagan E. Kelly, Jiacai Zhuo, Hengjun Chao. 265 Pre-Existing Immunity with Either Th1 or Th2 Predominance Inhibits Expression of Transgene in Skeletal Muscle of Immune Competent Animals An-Bang Liu. 266 Creating Retroviral Vector Encoding Two Homologous Chimeric Immune Receptors with Degenerated Codons Wen Yang, Li Lu, Jiangzhong Ma, Richard P. Junghans. Gene Modification 267 Gene Targeting Induced by Engineered Endonucleases Derived from the I-CreI Meganuclease Jean-Pierre Cabaniols, Sophie Leduc, Cécile Jacqmarcq, Christophe Perez, Julianne Smith, Sylvestre Grizot, Sylvain Arnould, Philippe Duchateau, Frédéric Pâques. 268 Transcriptional and Post-Translational Regulation System for Hypoxia Specific Gene Therapy Hyun Ah Kim, Cheol-Hee Ahn, Sung Wan Kim, Minhyung Lee. 269 Flow Cytometric Analysis of DNA Binding and Cleavage by Cell Surface-Displayed Homing Endonucleases Petra Volna, Jordan Jarjour, Sarah Baxter, Barry L. Stoddard, Raymond J. Monnat, Jr, Andrew M. Scharenberg. 270 Gene Transfer of an Engineered Transcription Factor Promoting Expression of VEGF-A Accelerates Rehabilitation of the Crushed Recurrent Laryngeal Nerve S. Brett Heavener, Kevin Fung, S. Kaye Spratt, Casey C. Case, Laurel E. Roberts, Norman D. Hogikyan, Eva L. Feldman. 271 Expression of IGF1 Receptor Ribozyme and IGFBP3 To Inhibit Rat Glioma Cell Growth Leah R. Villegas, Maria Grant, Sean M. Sullivan. 272 Incorporation of a Prostate-Specific Amplification Loop with FasL and TRAIL Expression To Boost Prostate Cancer Killing Jan Woraratanadharm, Brian D. Hoel, John Y. Dong. 273 Enhanced Hypoxia Specific Expression Activity of the Epo Enhancer and SV40 Promoter by Co-Transfection of the HIF- 1a Gene Suyeon Lee, Kyunghwa Kim, Hyun Ah Kim, Sung Wan Kim, Minhyung Lee. 274 Correction of Albinism Mutations by Targeted Genomic Rearrangements Using Engineered Zinc Finger Endonucleases Murray H. Brilliant, Todd D. Camenisch, David J. Segal. 70

72 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Gene Modification continued 275 Effect of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element (WPRE) on Transgene Expression Controlled by the Human Synapsin 1 Gene Promoter Following Adenoviral-Mediated Gene Delivery Yongxin Gao, Hongwei Li, Nan Jiang, Rachael Harrison, Colin Sumners. 276 Synthesis and Evaluation of Chemically Modified Peptide Nucleic Acids as Potential beta-globin IVS2 Gene-Targeting Agents Pallavi S. Lonkar, Jean Kuan, Peter M. Glazer. 277 A Highly Sensitive and Specific System for Large-Scale Breast Cancer Gene Expression Profiling Guohong Hu, Qifeng Yang, Honghua Li. 278 Conditionally Replicating Vector Mediated Transcriptional Silencing of HIV-1 Anne Marie-Turner, Peter Hawkins, Jiang Han, Kevin V. Morris. 279 A Threshold Exists for Expression of Short Hairpin RNA (shrna) in Liver Using Gutless Adenovirus Vectors Scott R. Witting, Matthew Brown, Romil Saxena, Nuria Morral. 280 Determination of an Induced Cellular Immune Profile Insights into Control of Viral Replication Jean D. Boyer, Gordon A. Vansant, Michele A. Kutzler, David A. Hokey, Mark G. Lewis, Maninder K. Sidhu, George B. Pavlakis, Barbara Felber, Joseph K. Montforte, David B. Weiner. 281 Characterization of FLP Recombinase Using Adenovirus Vector and Application for Gene Regulation in Mammalian Cells Saki Kondo, Yuki Takata, Yuzuka Takahashi, Yumi Kanegae, Izumu Saito. 282 A Quasi-Lentiviral Green Fluorescent Protein Reporter Exhibits Nuclear Export Features of Late Human Immunodeficiency Virus Type 1 Transcripts Marcus Graf, Christine Ludwig, Sylvia Kehlenbeck, Kerstin Junger, Ralf Wagner. 283 Constructoin of Artificial Promoters That Are Responstive to X-Ray Irradiation Ryohei Ogawa, Sung-il Lee, Go Kagiya, Hisao Hirano, Shigekazu Fukuda, Takashi Kondo, Tsutomu Kodaki. 284 Controlled Expression of an Inwardly-Rectifying Potassium Channel for Neuromodulation Qingshan Teng, Anand K. Katakam, Erin Carlton, Prasanna Kumar, Jun Yang, Thais Federici, Jonathan Riley, Dean Cress, Nicholas M. Boulis. 285 Construction and Testing of a Versatile One-Plasmid Regulated Expression System in Mice Using the Interferon beta Gene Paul Szymanski, Peter J. Kretschmer, Harald Petry, Alan Brooks, Hu Sheng Qian, Caralee Schaefer, Maxine Bauzon, Fang Jin, Ann Orme, Peiyin Wang, Gabor M. Rubanyi, Terry Hermiston, Richard N. Harkins. 286 In Vitro Studies on the Redirection of Human Growth Hormone Sorting Gabor Z. Racz, Naimh X. Cawley, Peng Y. Loh, Bruce J. Baum. 287 An Omega-Based Bacterial Selection System for Engineering Artificial Zinc Finger Proteins Marcus B. Noyes, Xiangdong Meng, Joseph C. McNulty, Scot A. Wolfe. 288 Structure-Based Redesign of the Dimerization Interface Reduces the Toxicity of Zinc Finger Nucleases Michal Szczepek, Vincent Brondani, Janine Büchel, Luis Serrano, David J. Segal, Toni Cathomen. 289 Influence of Donor DNA Design on Uninduced and Double- Strand-Break-Induced Gene Correction Andrew C. G. Porter, Rajani K. Vangala. Stem Cell I 290 Transduction of a Mammary Epithelial Stem Cell Population Via Early Gestational Intra-Amniotic Gene Transfer Jesse D. Vrecenak, Masayuki Endo, Philip W. Zoltick, Antonetta Radu, Alan W. Flake. 291 Phase 1 Human Trial of Autologous Mesenchymal Stem Cell Transplantation for the Treatment of Decompensated Cirrhosis Mahdi Mohamadnejad, Kamran Alimoghaddam, Maryam Bashtar, Mohamad Bagheri, Ardeshir Ghavamzadeh, Reza Malekzadeh. THURSDAY, MAY 31 71

73 Abstract Directory THURSDAY, MAY 31 Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Stem Cell I continued 292 Long-Term Lentiviral Vector-Mediated Gene Marking of the Human Hematopoietic System in Humanized Rag2 / γc / (RAG-hu) Mice and Applications for HIV-1 Gene Therapy Bradford K. Berges, Paul J. Christine, Leila K. Remling, Ramesh Akkina. 293 Suicide Gene-Mediated Conditional Removal of CNS Tumors- Derived from CNS Transplanted Embryonic Stem Cells Juyeon Jung, Lorraine F. Pierre, Ronald G. Crystal. 294 Cell Cycling Prior to Sex Cord Closure Places Male Germ Cells at Risk of Transduction Paul Park, Ferhat Ozturk, Joe Tellez, Esmail D. Zanjani, Graca Almeida- Porada, Christopher D. Porada. 295 The K5 Promoter Enhances Gene Expression in Skin Stem Cell Populations after Early Gestational Intra-Amniotic Gene Transfer Masayuki Endo, Phillip W. Zoltick, William H. Peranteau, Antoneta Radu, Nidal Muvarak, Mayumi Ito, Zaixing Wang, George Cotsarelis, Alan W. Flake. 296 Transient Gene Expression, Random Chromosomal Integration and Homologous Recombination in Cynomolgus Monkey Embryonic Stem Cells with Helper-Dependent Adenoviral Vectors Keiichiro Suzuki, Kouichi Hasegawa, Kaoru Mitsui, Emi Aizawa, Haruka Shiiba, Hirofumi Suemori, Norio Nakatsuji, Ko Mitani. 297 A Comparison of Transduction Efficiency and Long- Term Repopulating Capacity of Lentiviral Transduced SLAM Versus KSL-Enriched HSC Using an Abbreviated Transduction Protocol Pablo Laje, Nidal Muvarak, Philip W. Zoltick, Alan W. Flake. 298 Self-Renewal and Differentiation of Human Mesenchymal Stem Cells from Single Cell Clonal Assays C. Chang I. Lee, Joanne Huang, Jared E. Christensen, Christine I. Mall, Alyssa C. Leapley, Mervin C. Yoder, Alice F. Tarantal. 299 Hepatocyte Propagation In Vivo for Liver Tissue Engineering Kazuo Ohashi, Kohei Tatsumi, Miho Kataoka, Chise Tateno, Katsutoshi Yoshizato, Akira Yoshioka, Teruo Okano, Yoshiyuki Nakajima. 300 Development of Megakaryocyte-Specific Lentiviral Vectors for Gene Therapy of Platelet Disorders Liesbeth De Waele, Kathleen Freson, Veerle Gillijns, Chantal Thys, Chris Van Geet, Desire Collen, Thierry VandenDriessche, Marinee Chuah. 301 Mobilization in a Thalassemic Mouse Model: Implications for Gene Therapy of Thalassemia Evangelia Yannaki, Evangelia Athanasiou, Nikoletta Psatha, Garyfalia Karponi, Dimitris Bougiouklis, Varnavas Constantinou, Angeliki Xagorari, Panayotis Kaloyannidis, Ioannis Batsis, Achilles Anagnostopoulos, Athanasios Fassas. 302 Intraesophageal Manganese Superoxide Dismutase Plamsid/Liposome (MnSOD) Administration before Irradiation Increases Intravenous Engraftment of Esophageal Stem Cells Yunyun Niu, Michael W. Epperly, Hongmei Shen, Joel S. Greenberger. 303 Efficient Gene Transfer into Long-Term Repopulating Hematopoietic Stem Cells from Clinically Relevant Sources Martina Cesani, Alessia Capotondo, Eugenio Montini, Laura Tononi, Manfred Schmidt, Fabrizio Benedicenti, Anna Zingale, Francesca Santoni de Sio, Samantha Scaramuzza, Christof Von Kalle, Luigi Naldini, Alessandra Biffi. 304 A Transformed Cell Population Derived from Long Term Cultured Mesenchymal Stem Cells Has No Functional Effect after Transplantation into the Injured Heart Nan Ma, Wenzhong Li, Dario Furlani, Lee-Lee Ong, Karola Lützow, Christian Klopsch, Andreas Liebold, Andreas Lendlein, Gustav Steinhoff. 305 Surface Epitopes Involved in Cell Motility and Tumor Progression Are Expressed in Low Density Cultures but Not in Confluent Cultures of Human Multipotent Stromal Cells (hmscs) Ryang Hwa Lee*, Min Jeong Seo*, Andrey A. Pulin, Carl Gregory, Joni Ylostalo, Darwin J. Prockop. Cell Processing & Vector Production 306 Testing Large-Scale Lentiviral Vector Preparations for Replication Competent Lentivirus Lisa Duffy, Sue Koop, Jing Yao, Robert Getty, Scott Cross, Lakshmi Sastry, Kenneth Cornetta. 307 Generation of Lentivirus Vectors Using Recombinant Baculoviruses Hanna P. Lesch, Sanna Turpeinen, Einari A. Niskanen, Anssi J. Mähönen, Kari J. Airenne, Seppo Ylä-Herttuala. 72

74 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Thursday, May 31, 2007 Poster Session I continued 5:00 pm 8:00 pm Hall 4AB Cell Processing & Vector Production continued 308 Titer and Long Term Stability of Retroviral Titers Produced in Murine Based Producer Cell Lines Lisa Duffy, Sue Koop, Jing Yao, Kenneth Cornetta. 309 Plasmid DNA Production in a Wave Bioreactor under cgmp Conditions Kenneth A. Laderman, Jonathan M. Anderson, Ivy V. Derecho, Nina T. Dunphy, Ross J. McMahon, Valerie R. Quezada, David Hsu, Larry A. Couture. 310 A Rapid, Accurate and Precise Assay System To Analyze the Quality and the Quantity of Clinical Grade HSV Vector Stocks Ali Ozuer, Steve K. Wendell, Darren Wolfe, William F. Goins, David M. Krisky, Mohammad M. Ataai, Joseph C. Glorioso. 311 Development of a High Cell Density Process for cgmp Production of VRX496 Modified HIV Infected CD4+ T Lymphocytes Andrew Worden, Tony Encinas, Yelena Skripchenko, James Rogers, Reuben Cohen, Vladimir Slepushkin, Laurent Humeau. 312 Large Scale Lentiviral Vector Production in a GMP Facilty Lara J. Ausubel, Ken A. Laderman, Misty Shakeley, Anupriya Sharma, Sylvana Couture, Patricia Lopez, Christine Knoblauch, Jonathan Anderson, Ivy Derecho, Ross McMahon, David Hsu, Larry Couture. 313 Production of Ad/AAV-Hybrids Encoding Proteins That Inhibit Viral Propagation Gina Nichols, Tony Stepan, Kristin Parker, Eric J. Pastor, Richard W. Peluso, Barbara A. Thorne. 314 Retronectin Stimulation for the In Vitro Generation of Human Tumor-Reactive T Lymphocytes for Adoptive T Cell Transfer Therapy Seung Shin Yu, Emi Chatani, Kazuyuki Dan, Ikuei Nukaya, Hideto Chono, Junichi Mineno, Ikunosin Kato. 315 Optimization of Retrovirus Vector Mediated Gene Transfer Efficacy in the Closed System with the Combination of RetroNectin Pre-Loading and Low-Speed Centrifugation Hideto Chono, Ikuei Nukaya, Hirofumi Yoshioka, Hiromi Okuyama, Kazutoh Takesako, Junichi Mineno, Ikunoshin Kato. 316 The Human Papillomavirus E1 Gene Interferes with E2 and E6 Helper Functions for Adeno-Associated Virus Replication Hong You, Yong Liu, Nalini Agrawal, Raghu Muppidi, Jawahar L. Mehta, Paul L. Hermonat. 317 Refinement of Laboratory Protocols for AAVrh.10 Production into Production Batch Records Suitable for Production of Clinical Grade Vector Venus J. Williams, David S. Wei, Timothy P. O Connor, Dolan Sondhi, Neil R. Hackett, Ronald G. Crystal. 318 Engineering Polarized Epithelial Cells To Secrete Fluid Unidirectionally Senrong Qi, Most Nahid Parvin, Xibao Liu, Corrine M. Goldsmith, Takayuki Sugito, Changyu Zheng, Roy James Turner, Bruce Baum. 319 Inactivation of Herpes Simplex Viruses Following the Manufacturing of Recombinant Adeno-Associated Virus Vector by the Herpes Simplex Virus Co-Infection Method Marina M. Scotti, Jilin Liu, Guo-jie Ye. 320 Commissioning of an Aseptic Processing Area for Clinical Production of Adenoviral Vectors Maria Fe C. Medina, Uma Sankar, John Rudy, Jack Gauldie. Friday, June 1, 2007 Oral Abstract Session 300 RNA Vector Targeting, Expression and Integration 8:00 am 10:00 am Room :00 AM Efficient Gene Correction and Targeted Gene Addition in Human Stem Cells Using Engineered Zinc Finger Nucleases and Integrase-Defective Lentiviral Vectors Angelo Lombardo, Christian Beausejour, Pietro Genovese, Silvia Colleoni, Cesare Galli, Fyodor D. Urnov, Philip D. Gregory, Michael C. Holmes, Luigi Naldini :15 AM Transient Expression of WHIM-Type Mutant CXCR4 Human Hematopoietic Stem Cells Mediated by Integration Defective Lentivirus Vector Enhances Engraftment in the NOD/SCID Mouse Xenograft Model Toshinao Kawai, Uimook Choi, Po-Ching Liu, Larry M. Lantz, Harry L. Malech :30 AM Efficient Engraftment and In Vivo Selection of MGMT Gene- Modified Cells Following Nonmyeloablative Conditioning with BCNU and Temozolomide in the Dog Model Brian C. Beard, Christina Gooch, Laura J. Peterson, Hans-Peter Kiem. FRIDAY, JUNE 1 73

75 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Oral Abstract Session 300 continued RNA Vector Targeting, Expression and Integration 8:00 am 10:00 am Room :45 AM Promoter Analysis by Transduction of Fetal and Neonatal Mice by Lentiviral Vectors Steven J. Howe, Susannah Bailey, Nicola Brooke, Suzanne M. K. Buckley, Tristan R. McKay, Christine Kinnon, Adrian J. Thrasher, Charles Coutelle, Simon N. Waddington :00 AM LTR Attachment Site Mutant Non-Integrating Lentiviral Vectors Provide Greater Levels of Gene Expression Guiandre Joseph, Aparna Jasti, Lakshmi Sastry, Chunlu Guo, Eric Thompson, Michael Vasko, Kenneth Cornetta :15 AM Insertion of an Internal Intron Increases Transgene Expression Levels from a Bicistronic Lentivirus Vector Marcus Gentsch, Martin F. Ryser, Sebastian Brenner :30 AM Exploiting the Terminal Repeats of Lentiviral and Gammaretroviral Vectors To Block Both Transcriptional Readthrough and Enhancer Interactions Axel Schambach, Melanie Galla, Tobias Maetzig, Rainer Loew, Daniela Zychlinski, Christopher Baum :45 AM A Mechanistic Study of HIV-1 SIN Vector Mobilization To Address Unanticipated Biosafety Risks Brian J. Zeithaml, Hong Ma, Kafri Tal. Oral Abstract Session 301 Adenovirus Vectors: Tropism, Receptors and Targeting 8:00 am 10:00 am Room 4C1/4C :00 AM Role of the Factor X (FX) Light Chain in Adenovirus Binding and Serine Protease Exosite in Mediating Cell Transduction Alan L. Parker, John H. McVey, Simon N. Waddington, Oscar Lopez- Franco, Stuart A. Nicklin, Andrew H. Baker :15 AM An In Vivo Model To Evaluate Hepatic Targeting of Adenovirus by Coagulation Factor X Simon N. Waddington, John J. H. McVey, Alan L. Parker, Oscar Lopez- Franco, Suzanne M. K. Buckley, Stuart A. Nicklin, Andrew H. Baker :30 AM Comparison of Blood Interaction and Organ Biodistribution for Adenoviruses from Subspecies B, C, E and F Daniel Stone, Ying Liu, Zong-Yi Li, Andre Lieber :45 AM Alteration of Adenoviral Liver Tropism Following Hexon Protein Modification Frederic Vigant, Delphyne Descamps, Betsy Jullienne, Elisabeth Connault, Paule Opolon, Emmanuelle Vigne, Michel Perricaudet, Karim Benihoud :00 AM Converging Evidence for a Critical Role of the Diameter of Sinusoidal Fenestrae in Hepatocyte Transduction after Adenoviral Gene Transfer Frank Jacobs, Eddie Wisse, Yingmei Feng, Jan Snoeys, Joke Lievens, Changchun Ling, Hans Duimel, Désiré Collen, Peter Frederik, Bart De Geest :15 AM Efficient Gene Transfer in Cultured Aortic Smooth Muscle and Endothelial Cells by Magnetically Responsive Nanoparticles Impregnated with Adenovirus Michael Chorny, Robert J. Levy :30 AM Spliceform Specific Coxsackie-Adenovirus Receptor Interactions with MAGI-1b Nicholas D. Gansemer, Joseph Zabner, Katherine J. D. A. Excoffon :45 AM Protein IX-Modified Adenovirus Gene Transfer Vectors Can Efficiently Be Targeted to LRP by the Receptor-Associated Protein RAP Stephanie Corjon, Tatjana Engler, Stefan Kochanek, Florian Kreppel. Oral Abstract Session 302 DNA Vectors for Non-Viral Gene Therapy 8:00 am 10:00 am Room 608/ :00 AM Mutational Derivatives of PhiC31 Integrase with Increased Efficiency and Specificity Annahita Keravala, Solomon Lee, Bhaskar Thyagarajan, Eric C. Olivares, Vanessa E. Gabrovsky, Lauren E. Woodard, Michele P. Calos :15 AM Terminal Inverted Repeats of the Sleeping Beauty DNA Transposon Possess Residual Promoter-Like Activities Brian Moldt, Peter R. Andersen, Stephen R. Yant, Mark A. Kay, Jacob G. Mikkelsen :30 AM Autoregulation of Transposition of Sleeping Beauty Transposons by Ubiquitous N-Terminal, Dominant Negative Peptides Jeffrey M. Schreifels, Jason B. Bell, Elena L. Aronovich, David C. Erickson, R. Scott McIvor, Perry B. Hackett. 74

76 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Oral Abstract Session 302 continued DNA Vectors for Non-Viral Gene Therapy 8:00 am 10:00 am Room 608/ :45 AM Silencing of Episomal Transgene Expression in Liver by Plasmid Bacterial Backbone DNA Is Associated with Histone Modifications but Independent of CpG Methylation Efren Riu, Zhi-Ying Chen, Chen-Yi He, Hui Xu, Mark A. Kay :00 AM Exogenous Plasmid DNA Sequences Differentially Affect Episomal Transgene Silencing in Liver and Muscle Zhi-Ying Chen, Cheng-Yi He, Efren Riu, Mark A. Kay :15 AM CpGs Influence the Duration of Gene Expression from Plasmid Vectors after In Vivo Lung Delivery Anna E. Lawton, Lee A. Davies, Nelson S. Yew, Seng H. Cheng, Stephen C. Hyde, Deborah R. Gill :30 AM Deletion of CpG Motifs from Plasmid DNA for Sustained Transgene Expression In Vivo: Evaluation Based on a Statistical Analysis of the Expression Profile Makiya Nishikawa, Seiji Takemoto, Kiyoshi Yamaoka, Masaru Mitsui, Hisoon Lee, Yoshinobu Takakura :45 AM Development of S-MAR Plasmid Vectors for Persistent Expression and Maintenance, In Vivo Orestis Argyros, Suet-Ping Wong, Simon Waddingotn, Charles Coutelle, Andrew Miller, Richard P. Harbottle. Oral Abstract Session 303 Inborn Errors of Metabolism I 8:00 am 10:00 am Room 4C :00 AM A Hepatitis B Virus Protein X Phenylalanine Hydroxylase Fusion Protein Is Detected in Pahenu2 Mice Treated with AAV-WPRE Vectors Jennifer E. Embury, Philp J. Laipis :15 AM Insulin Replacement Gene Therapy for Type 1 Diabetes Eric C. Hsu, Timothy J. Kieffer, Scott Pownall, Yvonne Luu, Kathryn Burton, Alex Trinh, Michele P. Calos, Paul D. Robbins, Anthony T. Cheung :30 AM Therapeutic Effects of Bone Marrow Transplantation for Metachromatic Leukodystrophy Using HoxB4 Over- Expressing Cells Noriko Miyake, Koichi Miyake, Stefan Karlsson, Takashi Shimada :45 AM Biodistribution of AAV1-LPL S447X Vector Sequences after Intramuscular Administration to Lipoprotein Lipase Deficient Patients Melchior C. Nierman, Jaap Twisk, Wim T. Hermens, Andrew C. Bakker, Bossink Marieke, Sander J. van Deventer, Belia Rekke, John J. Kastelein, Erik S. Stroes, Jan Albert Kuivenhoven, Janneke J. Meulenberg :00 AM Very Few Vector Particles of a Small-Genome AAV Vector Prolong Survival in Glycogen Storage Disease Type Ia Mice and Dogs Dwight D. Koeberl, Sun Baodong, Carlos Pinto, Brown Talmage, Bird Andrew, Y.-T. Chen :15 AM Hematopoietic Stem Cell Based Gene Therapy for the Treatment of Metachromatic Leukodystrophy: Towards Clinical Testing Alessandra Biffi, Maria Sessa, Alessia Capotondo, Martina Cesani, Eugenio Montini, Laura Tononi, Sergio Marchesini, Manfred Schmidt, Christoff Von Kalle, Claudio Bordignon, Maria Grazia Roncarolo, Attilio Rovelli, Luigi Naldini :30 AM Aortic Disease in Mucopolysaccharidosis I Mice Is Due to Upregulation of Elastase Proteins and Can Be Prevented with Neonatal Gene Therapy in Mice and Dogs Xiucui Ma, Mindy Tittiger, Russ Knutson, Robert Mecham, Laura Schaller, Xu Zhang, Mark E. Haskins, Anne Traas, Meg Sleeper, Katherine P. Ponder :45 AM Correction of Hypoglycemia Following Gene Therapy in Dogs Affected with Glycogen Storage Disease Type Ia Carlos R. Pinto, Talmage T. Brown, Daniel M. Kozink, Amanda K. Demaster, Meghan A. Kruse, Kazuhiro Oka, Andrew Bird, Mark W. Jackson, Yuan-Tsong Chen, Lawrence Chan, Dwight D. Koeberl. Oral Abstract Session 304 Cardiovascular Gene Therapy 8:00 am 10:00 am Room 4C :00 AM Ad2-Mediated Expression of a Constitutively Stable Hypoxic Inducible Factor-1α Enhances Collateral Development and Reduces Vascular Leakage in a Diabetic Rat Model of Hindlimb Ischemia Adam J. Belanger, Hidetoshi Kajiwara, Zhengyu Luo, Akihiro Urabe, Seng H. Cheng, Seibu Mochizuki, Canwen Jiang. FRIDAY, JUNE 1 75

77 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Oral Abstract Session 304 continued Cardiovascular Gene Therapy 8:00 am 10:00 am Room 4C :15 AM Adenoviral enos-eluting Stents Result in Accelerated Re- Endothelialisation and a Reduction in Neointima Formation in an In Vivo Rabbiat Iliac Artery Stent Model Faisal Sharif, Sean O. Hynes, Ronan Cooney, Linda Howard, Jill McMahon, Kieran Daly, James Crowley, Frank Barry, Timothy O Brien :30 AM Novel Gene Transfer Vectors To Modulate the Renin Angiotensin System Stuart A. Nicklin, Anna F. Dominiczak, Andrew H. Baker :45 AM Use of Gene Transfer To Identify Cell and Tissue Specific Splicing Factors That Dictate Alternative Splicing of the Human VEGF Gene Rui Wang, Ronald G Crystal, Neil R. Hackett :00 AM Bone Marrow Cells Recruited through the Neuropilin-1 Receptor Are Essential To Promote Arterial Formation at the Sites of Adult Neoangiogenesis Serena Zacchigna, Lucia Pattarini, Lorena Zentilin, Silvia Moimas, Nikola Arsic, Sabrina Tafuro, Alessandro Carrer, Mauro Giacca :15 AM Helper-Dependent Adenovirus-Mediated LDLR Gene Therapy Reverses Hypercholesterolemia in LDLR-Deficient Rhesus Monkeys In Vivo Kazuhiro Oka, Charles E. Mullins, Mark A. Law, Rampratap P. Kushwaha, Karen Rice, Lawrence Chan :30 AM AAV Vector-Mediated Sustained Expression of Prostacyclin Synthase Ameliorates Obesity in Zucker Fatty Rats Takayuki Ito, Takashi Okada, Jun Mimuro, Masanori Nakata, Ryosuke Uchibori, Masashi Urabe, Hiroaki Mizukami, Akihiro Kume, Yoichi Sakata, Toshihiko Yada, Kazuyuki Shimada, Keiya Ozawa :45 AM Over Expression of the Angiotensin Type 2 Receptor in the Rostral Ventrolateral Medulla (RVLM) Decreases Basal Blood Pressure in Normotensive Rats Hongwei Li, Yongxin Gao, Erick Bourassa, Robert C. Speth, Colin Sumners. Oral Abstract Session 305 Gene Transfer to Protect from Infectious Agents and Their Products 8:00 am 10:00 am Room :00 AM Genetic Modification of T Cells To Prevent CMV Infection after Umbilical Cord Blood Transplantation Helen A. Andersson, Harjeet Singh, Juri G. Gelovani, Laurence J. N. Cooper :15 AM Prolonged and Liver Specific Expression of IL-12 Breaks Tolerance Against Viral Antigens and Induces a Marked and Sustained Antiviral Effect in Woodchucks with Chronic Viral Hepatitis Julien Crettaz, Pedro Berraondo, Laura Ochoa, Astrid Paneda, Itziar Otano, Stephan Kochanek, Juan Ruiz, Jesus Prieto, Gloria Gonzalez- Aseguinolaza :30 AM MicroRNA-Like Hairpin Sequences Capable of Inhibiting HBV Replication Efficiently In Vitro and In Vivo Abdullah Ely, Tanusha Naidoo, Steven M. Mufamadi, Carol Crowther, Patrick B. Arbuthnot :45 AM Inhibition of HIV-1 Replication with mirna-sirna Hybrids Expressed from RNA Polymerase Promoters Priscilla Yam, Hsin Lung Lo, Tammy Chang, Paola Marcovecchio, Shirley Li, John A. Zaia, Jiing-Kuan Yee :00 AM Combinatorial Gene Therapy To Inhibit HIV: Improving the Therapeutic Effect by Adding Transgenes Targeting Different Stages in the HIV-1 Replication Cycle Alena A. Chekmasova, Carmen N. Nichols, David S. Strayer :15 AM Novel AAV Vaccine Vector Circumvents Pre-Existing Immunity in Human Populations Jianping Lin, Luk H. Vandenberghe, Roberto Calcedo, Joanita M. Figueredo, James R. Miller, James M. Wilson :30 AM Preventing HIV-1 Tat-Induced Apoptosis Using Antioxidant Enzymes: Mechanistic and Therapeutic Implications Lokesh Agrawal, J.-P. Louboutin, David S. Strayer :45 AM Cytotoxic T Lymphocyte Therapy for the Treatment and Prevention of Adenovirus Infection after Stem Cell Transplant Ann M. Leen, Gary D. Myers, Anne Christin, Heidi Weiss, Adrian P. Gee, Kathryn Leung, Robert A. Krance, Malcolm K. Brenner, Helen E. Heslop, Cliona M. Rooney, Catherine M. Bollard. 76

78 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Oral Abstract Session 306 Cancer Immunotherapy: Receptor-Modified T Cells and T Cell Trafficking 8:00 am 10:00 am Room 606/ :00 AM Selecting Highly Affine and Well Expressed TCRs for Gene Therapy of Melanoma Annelies Jorritsma, Raquel Gomez, Maarten Dokter, Willeke van de Kasteele, Yvonne Zoet, Ilias Doxiadis, Nathalie Rufer, Pedro Romero, Rick Morgan, Ton Schumacher, John Haanen :15 AM Antigen Dose-Responsiveness and Immunoselectivity of CD8 + T Cells Bearing Chimeric T Cell Receptors Specific for CD20 and CD22: Implications for Immunotherapy of B Cell Malignancies Scott E. James, Michael C. Jensen, Yukang Lin, Philip D. Greenberg, Oliver W. Press :30 AM Lentivirally Engineered Primary Human Peripheral Blood Lymphocytes with Anti-CEA Chimeric Immune Receptor Efficiently Recognize and Lyse CEA + Tumor Cells Nachimuthu Chinnasamy, David E. Gilham, James Shaffer, Robert E. Hawkins, Dhana Chinnasamy :45 AM A First-in-Human Clinical Trial of Adoptive Therapy Using CD19-Specific Chimeric Antigen Receptor Re-Directed T Cells for Recurrent/Refractory Follicular Lymphoma Michael C. Jensen, Leslie Popplewell, David L. DiGiusto, Michael Kalos, Laurence J. N. Cooper, Andrew Raubitschek, Stephen J. Forman :00 AM Designer Cellular Immunotherapy: Zinc Finger Nucleases Generate Glucocorticoid Receptor Negative IL-13 Zetakine Expressing CD8+ T-Cells Andreas Reik, Michael C. Holmes, Yuanyue Zhou, Matthew Mendel, Pei- Qi Liu, Gary K. Lee, Nhu Tran, Philip Tam, David Paschon, Yanhong Kong, Edward J. Rebar, Dale Ando, Philip D. Gregory, Michael C. Jensen :15 AM Investigation of Therapeutic Immune-Responses to Cancer In Vivo: Tracking Lymphocytes with Transgenic T-Cell- Receptor Specific to a Melanoma Antigen by PET Imaging Richard C. Koya, Stephen Mok, Begonya Comin-Anduix, Caius Radu, Chengyi J. Shu, Pejman Kharazi, James S. Economou, Owen N. Witte, Antoni Ribas :30 AM Improved Homing of Antigen-Specific T Cells to Hodgkin s Tumor Cells by Forced Expression of CCR4 Receptor A. Di Stasi, G. Dotti, A. Foster, L. Zhang, H. M. Heslop, C. M. Rooney, M. K. Brenner, B. Savoldo :45 AM Phase 1/2a, Dose-Escalation, Safety and Proof of Concept Study of Intravesical BC-819 in Patients with Superficial Bladder Cancer Patricia Ohana, Suhail Ayesh, Tatiana Birman, Jennifer Galula, Ofer Gofrit, Abraham A. Sidi, Shalva Biniamin, Ilan Leibovitch, Moshe Shalev, Abraham Hochberg. Oral Abstract Session 307 Tumor Suppressor and Targeted Gene Therapy 8:00 am 10:00 am Room :00 AM Targeted Delivery of Interferon-α by Gene Modified Hematopoietic Stem Cells Inhibits Tumor Growth Michele De Palma, Ferdinando Pucci, Letterio S. Politi, Giovanni Sitia, Stefania Mazzoleni, Mary Anna Venneri, Luca G. Guidotti, Rossella Galli, Luigi Naldini :15 AM Enhanced Tumor Cell Killing in a Human Glioblastoma Xenograft Model with TRAIL Expressed from an Engineered Adenovirus Vector Martin E. Wohlfahrt, Brian C. Beard, André Lieber, Hans-Peter Kiem :30 AM Harnessing the P53 Pathway To Drive Viral Expression and Inhibit Tumor Growth Via Strategic Promoter/Transgene Combinations Bryan E. Strauss, Andre Giannetti, Anna Carolina V. de Carvalho, Christian A. Merkel, Marcio C. Bajgelman, Paula Fratini, Rafael Soares, Vinicius Melo, Eugenia Costanzi-Strauss :45 AM Clinical Experience with the mda-7/il-24 Tumor Suppressor Sunil Chada, John Nemunitais, Alex Tong, Elizabeth Grimm, Kevin Kim, Julie Ellerhorst, Robert E. Sobol, Evgeny Matyakin, Fagim Mufazalov, Dmitry Bentsion, Nina Krutilina, Konstantin Mardaleishvili, Kathy Hanchett, Julie Sicam, Ramesh Rajagopal, Kerstin Menander :00 AM Urokinase-Targeted Oncolytic Sendai Virus Vectors, Which Are Activated under Urokinase-Rich Environment, Selectively Kills Solid Tumor Via Syncytia Formation Hiroaki Kinoh, Makoto Inoue, Atsushi Komaru, Yasuji Ueda, Yoshikazu Yonemisu, Mamoru Hasegawa :15 AM Clinical Investigation of Bifunctional shrna Knockdown of Acid Ceramidase John J. Nemunaitis, Neil Senzer, Donald Rao, Phillip B. Maples, Xiang Liu, James Norris. FRIDAY, JUNE 1 77

79 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Oral Abstract Session 307 continued Tumor Suppressor and Targeted Gene Therapy 8:00 am 10:00 am Room :30 AM Efficient Targeting of AAV Vectors to Tumor-Associated Angiogenic Vasculature Following Intravascular Delivery Matthew D. Stachler, Jeffrey S. Bartlett :45 AM Telomerase-Specific Virotherapy Targeting Lymph Node Metastasis of Gastrointestinal Cancer Toru Kojima, Yuichi Watanabe, Yuuri Hashimoto, Ryo Sakai, Shinji Kuroda, Futoshi Uno, Shunsuke Kagawa, Yasuo Urara, Noriaki Tanaka, Toshiyoshi Fujiwara. Oral Abstract Session 320 AAV Vector Biology 1:15 pm 2:45 pm Room :15 PM Nox2-Dependent Endosomal H 2 O 2 Facilitates raav2 Infection by Activating Capsid PLA 2 Liang N. Zhang, Justin Schwartz, Nicholas Keiser, Ziying Yan, Yulong Zhang, Diana C. M. Lei-Butters, John F. Engelhardt :30 PM AAV Exploits Conditions of Cellular Stress Jarrod S. Johnson, R. Jude Samulski :45 PM Sequencing of raav Genome Termini in Wild Type and SCID Mouse Tissues Unravels a Role of Artemis/DNA-PKcs in Opening AAV-ITR Hairpins In Vivo Katsuya Inagaki, Congrong Ma, Theresa A. Storm, Mark A. Kay, Hiroyuki Nakai :00 PM Adeno-Associated Virus 2-Mediated Gene Transfer: Tyrosine-Phosphorylation of Capsid Proteins and Its Consequences on Transgene Expression Li Zhong, Baozheng Li, Sergei Zolotukhin, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, Arun Srivastava :15 PM Subcellular Trafficking of Recombinant AAV: Nucleolar Accumulation of Capsids Is Unique to Infectious Virions Jarrod S. Johnson, R. Jude Samulski :30 PM Involvement of Growth Factor Receptors in Heparin Mediated Enhanced Transduction of AAV3b Based Vectors Emily L. Messina, Dawn E. Bowles. Oral Abstract Session 321 HSV and Other DNA Viruses 1:15 pm 2:45 pm Room 4C :15 PM Long-Term Regulated Functional Complementation of LDLR Deficiency in Cells Using ibac-smar-ldlr, a Novel Infectious Genomic DNA Episomal Vector Michele M. P. Lufino, Olivia C. Hibbitt, Richard Wade-Martins :30 PM Robust Induction of Type I Interferons Immediately after Systemic Delivery of HSV Amplicon Vectors Elicits Infiltration of Immune Cells and Suppresses Transcription of the Vector-Encoded Transgene Masataka Suzuki, Yoshinaga Saeki :45 PM Targeting of Metabolically Biotinylated Baculoviruses Minna U. Kaikkonen, Jenni I. Viholainen, Jani K. Räty, Ale Närvänen, Seppo Ylä-Herttuala, Kari J. Airenne :00 PM Neuroblastoma-Derived Tumorspheres with Cancer Stem Cell-Like Properties Are Sensitive to a Transcriptionally Targeted Oncolytic Herpes Simplex Virus Yonatan Y. Mahller, William H. Baird, Jon P. Williams, Jianqiang Wu, Yoshinaga Saeki, Jonathan Grossheim, Margaret H. Collins, Jose A. Cancelas, Nancy Ratner, Timothy P. Cripe :15 PM Monitoring Sarcoma Response to Systemic Gene Therapy with a Targeted Adeno-Associated Virus/Phage: AAVP Amin Hajitou, Suren Soghomonyan, Jonathan Hannay, Mian Alauddin, Juri G. Gelovani, Raphael E. Pollock, Dina Lev, Renata Pasqualini, Wadih Arap :30 PM A Tetracycline Regulatable Oncolytic HSV-1 Recombinant for Tumor Therapy Feng Yao, Oliver Bleiziffer, Nao Murakami, Ximing Xu, Richard Brans. Oral Abstract Session 322 Non-Viral Delivery Strategies 1:15 pm 2:45 pm Room 4C1/4C :15 PM Expression and Distribution of Compacted-DNA Nanoparticles in the Developing Retina Shannon M. Conley, Mark J. Cooper, Muna I. Naash :30 PM Non-Viral Gene Delivery to the Primate CNS: A Model for Pre-Operative Neuroprotection James G. Hecker, Michael H. Nantz, Alice F. Tarantal. 78

80 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Oral Abstract Session 322 continued Non-Viral Delivery Strategies 1:15 pm 2:45 pm Room 4C1/4C :45 PM Transposition of Anti-HIV Transgenes rhtrim5α, mapobec 3, Anti-CXCR4 and CCR5 sirnas by Sleeping Beauty (SB) Transposon System for HIV Gene Therapy Mayur C. Tamhane, Ramesh Akkina :00 PM Mechanism for Difference in the Transcription Activity of Nucleus-Delivered Transgene Introduced by Adenovirus and Lipoplex Hidetaka Akita, Susumu Hama, Hiroyuki Mizuguchi, Hideyoshi Hatashima :15 PM Binding of Oligodeoxynucleotides to the beta-(1->3)-d- Glucan Schizophyllan Increases Their Stability in Blood and Potentiates the Efficacy of Antisense Therapy In Vivo Yoshikazu Koyama, Shigeki Ohshima, Shin Onodera, Tatsuya Ohkawara, Jun Nishihira, Yoshifumi Takei, Susumu Sato, Kazunori Asada, Atsushi Uno, Seiji Shinkai, Shuichiro Uehara, Yoichi Takeda, Naohiko Shimada, Kazuo Sakurai :30 PM Comparison of Intravesical Interleukin-12 Gene Therapy and Intravesical BCG Therapy in an Orthotopic Bladder Cancer Model Minoru Horinaga, Kelley Harsch, Ryuichi Fukuyama, Warren Heston, William Larchian. Oral Abstract Session 323 Inborn Errors of Metabolism II 1:15 pm 2:45 pm Room 608/ :15 PM A Novel In-Vivo Approach for AAV-Mediated Gene Therapy of Hemophilia B Using Salivary Glands as a Depot Organ Hiroshi Takahashi, Bruce Baum, Jay Chiorini :30 PM Partial Correction of the FVIII Deficit in Hemophilia A Mice Using Minicircle DNA Vectors Expressing Pre-Trans-Splicing Molecules S. Gary Mansfield, Ping Du, Suja Hiriyanna, M. Puttaraju, Gerard J. McGarrity, Mariano A. Garcia-Blanco :45 PM A microrna-regulated Lentiviral Vector Mediates Stable Correction of Hemophilia B Mice Brian D. Brown, Alessio Cantore, Lucia Sergi Sergi, Patrizia Della Valle, Armando D Angelo, Luigi Naldini :00 PM Long-Term Phenotypic Correction of Murine Hemophilia A and Immunological Differences of Bioengineered FVIII Variants Delivered by Helper-Dependent Adenoviral Vectors Vincenzo Cerullo, William M. McCormack, Michael Seiler, Viraj Mane, Christian Clarke, Steven W. Pipe, Randal J. Kaufman, Brendan Lee :15 PM A Clinically-Relevant Gene Therapy Strategy for Hemophilia A: Non-Myeloablative Transplantation of Hematopoietic Stem Cells Transduced with a High-Expression Factor VIII Transgene Lucienne M. Ide, Bagirath Gangadharan, Christopher B. Doering, H. Trent Spencer :30 PM Correction of the Factor VIII Deficiency in Hemophilia A Mice with Pre-Existing Anti-Factor VIII Inhibitors by Transplantation of Genetically-Modified Hematopoietic Stem Cells Expressing Porcine Factor VIII Christopher B. Doering, Bagirath Gangadharan, Lucienne M. Ide, Hillary Z. Dukart, H. Trent Spencer. Oral Abstract Session 324 Gene Therapy for the Musculo Skeletal System 1:15 pm 2:45 pm Room 606/ :15 PM Microdystrophin Expression in Mice Reveals Novel Roles for Dystrophin at the Myotendinous and Neuromuscular Junctions Glen B. Banks, Luke M. Judge, Paul Gregorevic, Sheng Li, Cailtin Doremus, Leonard Meuse, Eric E. Finn, James M. Allen, Stanley C. Froehner, Jeffrey S. Chamberlain :30 PM In Utero Delivery of Adenovirus Encoded TGF-β3 Rescues Cleft Palate in a TGF-β3 Knockout Mouse Ryan M. Spivak, Masayuki Endo, Philip W. Zoltick, Allison Zajac, Brian U. Ang, Alan W. Flake, Richard E. Kirschner, Hyun-Duck Nah :45 PM Amelioration of Arthritis Following Intra-Articular Administration of an Adeno-Associated Virus Type 5 Expressing Interferon-β under Control of a Disease Inducible Promoter Janik Adriaansen, Frits J. Fallaux, Carin J. de Cortie, Margriet J. B. M. Vervoordeldonk, Paul P. Tak. FRIDAY, JUNE 1 79

81 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Oral Abstract Session 324 continued Gene Therapy for the Musculo Skeletal System 1:15 pm 2:45 pm Room 606/ :00 PM Phase I-IIa Gene Therapy Protocol for LGMD 2C: Clinical Strategy and Implications Hafedh Haddad, Didier Caizergues, Aude Rigolet, Carole Masurier, Anne- Marie Douar, Bernard Gjata, Patricia Noguiez-Hellin, Muriel Audit, Loic Millot, Otto Merten, Pascal Laforet, France Leturcq, Thomas Voit, Bruno Eymard, Pierre Carlier, Olivier Benvéniste, François Lemoine, Norma Romero, David Klatzmann, Jean-Yves Hogrel, Marie Rosier-Montus, Patrice Denèfle, Serge Herson, Anne-Marie Masquelier :15 PM AAV-Mediated Gene Targeting of COL1A2 Exon 2 in Mesenchymal Stem Cells from Individuals with Osteogenesis Imperfecta David R. Deyle, Ulrike Schwarze, Joel R. Chamberlain, Peirong Wang, Yi Li, Peter H. Byers, David W. Russell :30 PM Sustained Efficacy through Adeno-Associated Virus Vector- Mediated Gene Replacement in Advanced Pompe Disease Baodong Sun, Haoyue Zhang, Andrew Bird, Sarah P. Young, Dwight D. Koeberl. Oral Abstract Session 325 Lung and Respiratory Disease 1:15 pm 2:45 pm Room :15 PM AAV6.2: An Efficient and Safe Gene Therapy Clinical Candidate for the Treatment of Cystic Fibrosis Airway Disease Maria P. Limberis, Luk H. Vandenberghe, Liqun Zhang, Raymond J. Pickles, James M. Wilson :30 PM Significant Airway Transduction after In Utero Administration of a Gp64 Pseudotyped Lentiviral Vector Suzanne M. K. Buckley, Steven J. Howe, Vicky Sheard, Donald S. Anson, David W. Parsons, Adrian J. Thrasher, Charles Coutelle, Simon N. Waddington, Tristan R. McKay :45 PM Bronchoscope-Guided, Targeted Lobar Aersolization of HDAd into Nonhuman Primate Lungs Results in Uniform, High Level Pulmonary Transduction, Long Term Transgene Expression and Negligible Toxicity Peter Hiatt, Nicola Brunetti-Pierri, Ruth McConnell, Donna Palmer, Yu Zuo, Milton Finegold, Arthur Beaudet, Philip Ng :00 PM Lung-Directed Gene Therapy for Human Alpha-1 Antitrypsin Deficiency Using AAV6 Vectors Christine L. Halbert, Andrew E. Vaughan, A. D. Miller :15 PM Correction of CFTR Function by Segmental Trans-Splicing Yuhu Song, Howard H. Lou, John Ang, Julie L. Boyer, Philip L. Leopold, Ronald G. Crystal :30 PM Persistent Gene Expression in the Ovine Lung from a Human Elongation Factor 1 Alpha Promoter Plasmid Following Non- Viral Gene Delivery Gerry McLachlan, Catherine Gordon, Christina Vrettou, Eilidh Baker, Peter Tennant, Alison Baker, Rebecca L. Coles, Ian A. Pringle, Steve C. Hyde, Deborah R. Gill, D. David S. Collie, Lee A. Davies. Oral Abstract Session 326 Stem Cells Translational Technology Session 1:15 pm 2:45 pm Room :15 PM Recruitment by the Liver of Genetically Modified Endothelial Progenitor Cells in Models of Acute and Chronic Liver Damage Milosz P. Kawa, Verónica Fernández-Ruiz, Guanhua Yang, Ignacio Herrero, Cheng Qian, Jesús Prieto :30 PM Changing the HLA Type of Human Embryonic Stem Cells Roli K. Hirata, Yunwen Huo, Angel Nelson, Maja Salva, Carol Ware, David W. Russell :45 PM Magnetic Resonance Imaging Reveals Hematopoietic Stem/ Progenitor Cells Homing to the Brain of Mice Affected by Metachromatic Leukodystrophy Alessia Capotondo, Letterio S. Politi, Angela Gritti, Angelo Quattrini, Giuseppe Scotti, Luigi Naldini, Alessandra Biffi :00 PM Sustained Engraftment of MGMTP140K Gene-Modified Dog Hematopoietic Cells in Primary and Secondary Recipients Brian C. Beard, Kirsten Keyser, Reeteka Sud, Sabine Gerull, Laura J. Peterson, Tobias Neff, Grant D. Trobridge, Hans-Peter Kiem :15 PM Production of Transgenic Cloned Domestic Cat Embryos by Lentivirus-Mediated Transgenesis David M. Ricks, Martha C. Gomez, Robert H. Kutner, C. Earle Pope, Betsy L. Dresser, Jakob Reiser. 80

82 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Oral Abstract Session 326 continued Stem Cells Translational Technology Session 1:15 pm 2:45 pm Room :30 PM Highly-Efficient Genome Modification in Human Mesenchymal Stem Cells Using Engineered Zinc Finger Nucleases Shuyuan Yao, Jianbin Wang, Gary Lee, Aleida Perez, Nathaniel Wang, Kenneth Kim, James Li, Fyodor D. Urnov, Philip D. Gregory, Michael C. Holmes. Oral Abstract Session 327 Cell Processing and Vector Production 1:15 pm 2:45 pm Room 4C :15 PM Validation of a Large-Scale Semi-Closed System for the Manufacture of Autologous T Cells Genetically Targeted to the B Cell-Specific Antigen CD19 for a Phase I Trial in Patients with Chronic Lymphocytic Leukemia Daniel Hollyman, Jolanta Stefanski, Mark Przybylowski, Shirley Bartido, Oriana Borquez-Ojeda, Vanessa Capacio, Katherine Smith, James Hosey, Clare Taylor, Renier Brentjens, Michel Sadelain, Isabelle Riviere :30 PM Development of a Safe High-Titer Packaging System Based upon FeLV Raymond T. Doty, A. Dusty Miller, Jing Chen, Janis L. Abkowitz :45 PM Development of a cgmp Production Process for raav1 Vectors Produced Via Baculovirus Expression Kathyrn A. Rizzo, Robert A. Ballinger, Karl Anderson, Joseph A. Rininger :00 PM Engineering of Packaging Cell Lines for the Biomanufacturing of Retroviral Vectors Karim Ghani, Amine Kamen, Manuel Caruso :15 PM Large-Scale Production of Lentiviral Vectors Using Adherent and Suspension-Adapted Cells James P. Brady, Matthew Malehorn, Joseph C. Fratantoni, Linda N. Liu, Madhusudan V. Peshwa :30 PM Process Characterization and GMP Manufacture of a Gene Transfer Vector for Leber Congenital Amaurosis Bernd Hauck, Xingge Liu, Sonali Joyce, Alex Tai, Jeannette Bennicelli, Olga Zelenaia, Katherine A. High, J. Fraser Wright. Presidential Symposium 3:00 pm 5:15 pm Ballroom ABC 433 3:59 PM Cone-Targeted Gene Therapy by AAV John J. Alexander, Jijing Pang, Shannon E. Boye, Sanford L. Boye, Wentao Deng, SeokHong Min, Yukimo Unimo, Drew Everhart, Robert Barlow, Norman L. Hawes, Bo Chang, William W. Hauswirth :18 PM MicroRNA-Regulated Lentiviral Vectors Drive Robust Tissue-, Lineage- and Developmental Stage-Specific Gene Expression in Hematopoietic, Neural and Embryonic Stem Cells Bernhard Gentner, Brian D. Brown, Silvia Colleoni, Angela Gritti, Cesare Galli, Luigi Naldini :37 PM CNS Viral Vector Delivery under Real-Time Imaging Control Krystof S. Bankiewicz, Vanja Varenika, Piotr Hadaczek, John Forsayeth, William J. Boweres, Howard J. Federoff :56 PM Targeted Disruption of CCR5 Using Engineered Zinc Finger Protein Nucleases Provides In Vivo Protection from HIV Elena E. Perez, Jianbin Wang, Olga Liu, Kenneth Kim, Nathaniel Wang, Gary Lee, Jeffrey C. Miller, Dmitry Guschin, Victor Bartsevich, Dale Ando, Philip D. Gregory, James L. Riley, Michael C. Holmes, Carl H. June. Poster Session II 5:15 pm 8:15 pm Hall 4AB Adenovirus Vectors: Applications 437 Optimization of Capsid-Incorporated Antigens for an Improved Adenovirus Vaccine Approach Qiana L. Matthews, PingAr Yang, Qi Wu, Natalya Belousova, Angel A. Rivera, Mariam A. Stoff-Khalili, Reinhard Waehler, Hui-Chen Hsu, John Mountz, Hongju Wu, David T. Curiel. 438 Enhancement of Therapeutic Effect with Replication- Competent Retrovirus Vectors Using Helper-Dependent Adenovirus Shuji Kubo, Kazunori Haga, Akinobu Gotoh, Noriyuki Kasahara. 439 Promoter Control of Gene Expression in Adenovectors Delivered to the Vestibular System Hinrich Staecker, Mark Praetorius, Chi Hsu, Kim Baker, Peter Plinkert, Douglas E. Brough. 440 A New Infectivity-Enhanced Delta-24 Oncolytic Adenovirus with Capsid-Incorporated Dual Imaging Modality Reporter for a Dynamic Fluorographic and SPECT/PET-Based Imaging Anton V. Borovjagin, Mert Icyuz, Jing Li, Ronald D. Alvarez, David T. Curiel. FRIDAY, JUNE 1 81

83 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Adenovirus Vectors: Applications continued 441 Properties of Human Dendritic Cells after High Titer Infection with Oncolytic Adenoviruses Stephan Schierer, Ina Muller, Andrea Hesse, Jan Dorrie, Niels Schaft, Alexander Steinkasserer, Gerold Schuler, Eckhart Kampgen, Dirk M. Nettelbeck. 442 Examination of the Therapeutic Potential of an Engineered VEGF-A Transcription Activator in a Spinal Cord Injury Model Yang Liu, S. Kaye Spratt, Gary Lee, Michael G. Fehlings. 443 Systemic Dissemination of the Replication-Competent Oncolytic Adenovirus Telomelysin (OBP-301) Following Intratumoral Injection Yu-An Zhang, Alex W. Tong, Padmasini Kumar, Michael Nemunaitis, Minal Barve, Neil Senzer, Hitoshi Kawamura, John J. Nemunaitis. 444 YB-1 as a Target for the Development of Oncolytic Adenoviruses Klaus Mantwill, Regina Holzmueller, Moritz Widmaier, Emanuel Rognoni, Per S. Holm. 445 Effects of Exogenous p53 on Potency and Safety of Oncolytic Adenovirus Victor W. van Beusechem, Petra B. van den Doel, Juliette C. Blokker, Winald R. Gerritsen. 446 Antiviral Activity of Cidofovir Against Telomerase-Specific Replication-Competent Adenovirus, Telomelysin (OBP-301) Masaaki Ouchi, Hitoshi Kawamura, Katsuyuki Nagai, Yasuo Urata, Toshiyoshi Fujiwara. 447 Survivin-Responsive Conditionally Replicating Adenovirus Additionally Regulated with Cancer-Specific Promoter Achieved More Strict Cancer Specificity without Reducing Anti-Cancer Effect Yoshiteru Murofushi, Junichi Kamizono, Ngin Cin Khai, Tomoyuki Takahashi, Setsuro Komiya, Ken-ichiro Kosai. 448 Adeno-hTERT-E1A, a Highly Oncolytic, htert Promoter- Regulated Replication-Conditional Adenovirus Joshua C. Doloff, David J. Waxman, Youssef Jounaidi. 449 Effect of Preimmunization and Transient Immunosupression with CsA in Hepatic Adenovector Transduction in Cirrhotic Rats Ana S. Sandoval, Mayra Mena, Ana Marquez, Jesus Garcia-Bañuelos, Adriana Salazar, Juan Armendariz-Borunda, Estuardo Aguilar-Cordova. 450 Further Study of Gene Transduction into a Subset of Rat Dendritic Cells (DC) Generated from a Long Term Culture of Bone Marrow Cells: Apparent Resistance of Plasmacytoid DC to Gene Transduction by Adenoviral Vectors Ryota Haba, Atsushi Tsuji, Xiao-Kang Li, Naoko Fuji-Funeshima, Iwao Nozawa, Hiromitsu Kimura. 451 Generation of Apolipoprotein A-I Adenoviral Vectors Joshua M. Buckler, Anton S. McCourtie, Kun Qian, Shan Wen, David A. Dichek. 452 Effect of TBRII cyt Transduction in Pro-Fibrogenic Gene Expression in Activated Hepatic Stellate Cells Ana Marquez, Ana Sandoval, Jaime Gonzalez, Juan Armendariz-Borunda. 453 Rapid Identification and Classification of Adenoviral Serotypes by Mass Spectrometry Edward C. Nwanegbo, L. J. Sparvero, Andrew Amoscato, Jian Zhang, Kaori Okadah, Andra Gambotto. 454 Adenylyl Cyclase Type VI Regulates Akt Activity through a PH Domain Leucine-Rich Repeat Protein Phosphatase Mei Hua Gao, Tong Tang, Tracy Guo, Alexandra C. Newton, H. Kirk Hammond. 455 Plasmin Has an Important Role in Regulate Profibrogenic Molecules in HSC Miriam Ruth M. R. Bueno-Topete, Abril A. Martinez-Rizo, Jaime J. Gonzalez-Cuevas, Juan J. Armendariz-Borunda. 456 An In-Vitro Assay To Assess Secreted Osteogenic Factors from Genetically Engineered Cells Manjula Viggeswarapu, Maggie Bargouti, Chantrice Rogers, Louisa F. Titus, Scott D. Boden. 457 Replicative Adenovirus with Enhanced Tumor Specificity and Oncolysis Brian D. Hoel, Semyon Rubinchik, Min Luo, John Y. Dong. 82

84 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB HSV and Other DNA Viruses 458 Hexamethylene Bisacetamide Addition during Vector Packaging Leads to Reduced Helper Virus-Free HSV-1 Amplicon Expression Titers Via Suppression of Particle- Associated ICP0 Clark A. Burris, Suresh de Silva, Wade C. Narrow, Ann E. Casey, Louis T. Lotta, Jr, Howard J. Federoff, William J. Bowers. 459 The HSV-2 Protein ICP10PK Protects PC12 Cells from MPP+- Induced Apoptosis Jennifer M. Laing, Samantha Q. Wales, Laure Aurelian. 460 Imaging Transcriptional Activation of Gliomas in the Indicator Infectious BAC (iibac) of SPARC Hiroshi Nakashima, Yoshinaga Saeki, Antonio E. Chiocca. 461 Functional Genomic Analysis of Oncolytic HSV Infection in Human Cancer Cells Yonatan Y. Mahller, Bhuvana Sakthivel, Bruce J. Aronow, Ya-Hsuan Hsu, Ruty Mehrian-Shai, Timothy P. Cripe. 462 Minimal Risk of Herpes Simplex Virus Latency Following Intravenous or Intracerebral Injection of an ICP6-Deleted Oncolytic HSV Expessing CYP2B1 (rrp450) Is Not Increased by Cyclophosphamide Co-Administration Rebecca A. Gillespie, Mark A. Currier, Nancy M. Sawtell, Hiro Kambara, E. A. Chiocca, Timothy P. Cripe. 463 Generation of HSV Vectors Using a Highly Efficient BAC Homologous DNA Recombination Lv Miao, Fraefal Cornel, Kian Chuan Sia, Ivy A. W. Ho, Grace Y. Wang, Kam M. Hui, Paula Y. P. Lam. 464 SPECT/CT Imaging of Baculovirus Biodistribution in Rat Shows Kidneys To Be a Promising Target for Intraperitoneal Gene Delivery Jani K. Räty, Timo Liimatainen, Tuulia Huhtala, Minna U. Kaikkonen, Kari J. Airenne, Juhana M. Hakumäki, Ale Närvänen, Seppo Ylä- Herttuala. 465 Baculovirus Vectors Displaying Tumor-Homing Peptides: Binding and Entry Kinetics in Human Cancer Cells Anna R. Mäkelä, Johanna Rinne, Maija Vihinen-Ranta, Christian Oker- Blom. 466 Antiviral Effect Caused by Baculovirus Transduction Hampers Subsequent Transgene Expression in Rat Articular Chondrocytes Hsiao-Ping Lee, Tung-Hung Wang, Heng-Chun Shen, Chi-Yuan Chen, Yu-Chen Hu*. 467 Baculovirus-Induced Intranuclear Alterations in Human Cells Johanna Rinne, Minna Kaikkonen, Paula Ronkainen, Einari A. Niskanen, Teemu I. Ihalainen, Seppo Ylä-Herttuala, Markku S. Kulomaa, Kari J. Airenne, Maija Vihinen-Ranta. 468 A 96-Well Baculovirus Generation Platform for High- Throughput Gene Delivery Hanna-Riikka A. Karkkainen, Hanna P. Lesch, Antti I. Maatta, Anssi J. Mahonen, Miia M. Roschier, Pyry I. Toivanen, Kari J. Airenne, Olli H. Laitinen, Seppo Yla-Herttuala. 469 SV40 Vectors Expressing Insulin-Like Growth Factor I Revert Liver Cirrhosis in Rats Luciano Sobrevals, Astrid Pañedas, Nerea Razquin, Nerea Juanarena, Iñaki Monreal, Cristina Olagüe, Gloria Gonzalez-Aseguinolaza, Carlos Rodriguez, Jorge Quiroga, Jesús Prieto, Puri Fortes. 470 Aerosol Delivery of Lentivius-CTMP Suppressed Lung Cancer Growth through Controlling Akt-Related Signals in Lungs of K-ras Null Mice Soon Kyung Hwang, Myung-Haing Cho. 471 Baculovirus as a New Gene Delivery Vector for Stem Cells Engineering and Bone Tissue Engineering Ching-Kuang Chung, Li-Yu Sung, Shiaw-Min Hwang, Yu-Chen Hu. 472 Modulation of Microglial Cells towards a Neuroprotective Profile Using the RR Vector To Deliver the HSV-2 Protein ICP10PK Jennifer M. Laing, Laure Aurelian. FRIDAY, JUNE 1 83

85 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB DNA Vectors for Non-Viral Gene Therapy 473 Genetic Restoration of Ataxia Telangiectasia Cells Using a Human Artificial Chromosome Mediated-Genomic Cloning System Yasuhiro Kazuki, Hidetoshi Hoshiya, Yoshiteru Kai, Satoshi Abe, Masato Takiguchi, Mitsuhiko Osaki, Yuichi Yuichi Iida, Eiji Mizutani, Naoyo Kajitani, Toko Yoshino, Yasuaki Shirayoshi, Nozomi Tomimatsu, Masaharu Hiratsuka, Akihiro Kurimasa, Teruhiko Wakayama, Mitsuo Oshimura. 474 Smooth Muscle-Specific Non-Viral Gene Therapy Using Plasmid Vectors Aaron M. Miller, David A. Dean. 475 Minicircle Plasmid Containing the Human Manganese Superoxide Dismutase (MnSOD) Transgene Confers Radioprotection to Cells In Vivo Xichen Zhang, Michael W. Epperly, Mark A. Kay, Zhi-Ying Chen, Tracy Smith, Darcy Franciola, Joel S. Greenberger. 476 Group II Intron Mediated Gene Therapy Jacoba G. Slagter-Jager, Bruce A. Sullenger. 477 Effects of Hypoxia Response Element and DNA Targeting Sequence in VEGF Gene Expression in Hypoxia and Normoxia Chester B. Sacramento, Jane Z. Moraes, Rodrigo Tambellini, Sang W. Han. 478 Development and Production of Marker-Free Non-Viral Vectors Daniela Keller, Hans Huber, Juergen Marhofer, Irene Pfaffenzeller, Reinhard Grabherr. 479 Antisense Oligonucleotide Selection for Exon Skipping by a Readout Reporter System with High Capacity and Specificity Allen E. Zillmer, Qi L. Lu, Jeffrey Rosenfeld. 480 Designing a Targeted Plasmid DNA in Absence of Synthetic Vectors Feng Liu, Takuro Niidome, Yoichi Negishi, Leaf Huang. 481 Structure-Based Prediction of Insertion-Site Preferences into Chromosomes of Vectors Used for Gene Therapy Perry B. Hackett, Christopher S. Hackett, Aron M. Geurts. 482 Construction and Testing of a Liver-Directed Targeting Tether for Tissue-Specific Delivery of Sleeping Beauty Transposons Erik R. Olson, Jeffrey M. Schreifels, Jason B. Bell, R. Scott C. McIvor, Perry B. Hackett. 483 Phage C31 Integrase Induced Chromosomal Rearrangements in Normal Human Fibroblasts Involves DNA Damage Response Jian Liu, Torben Gjetting, Thomas G. Jensen. 484 Safety of ϕc31 Integrase Expression in Mouse Liver Following Hydrodynamic Injection Christopher L. Chavez, Lauren E. Woodard, Annahita Keravala, Vanessa Gabrovsky. 485 Analysis of the Transfection Behaviour of Supercoiled Plasmid DNA Concatemers In Vitro Christof Maucksch, Florian Hoffmann, Martin Schleef, Manish K. Aneja, Markus Elfinger, Dominic Hartl, Carsten Rudolph. 486 Rapid Deployment Plasmid Production Process, Combining Inducible High Yield Fed-Batch Fermentation Process with Novel Autolytic Plasmid DNA Purification Aaron Carnes, James Williams, Jeremy Luke, Sarah Langtry, Clague Hodgson. 487 Amplified IL-15 Expression Vector for Cancer Immuno-Gene Therapy Xianghui He, Weidong Li, Na Zhao, Yujie Qiu, Liwei Zhu. Physical Methods of Delivery I 488 Hydrodynamics and Ultrasound-Based Transfection of Heart with Plasmid DNA Leonardo P. Carvalho, Daniela Takeshita, Bruno Carillo, Bianca C. Lisboa, Abram Beutel, Eduardo Yasumura, Gustavo C. Molina, Valderez B. Valero, Ruy R. Campos, Hans F. Dohmann, Sang W. Han. 489 Temporal Expression Patterns of Various Growth Factors during the Cutaneous Wound Healing of Streptozotocin Diabetic Mice Provoked by Non-Viral VEGF 165 Gene Therapy Via Sonoporation Jeonghyun Park, Changshin Yoon, Hyesook Chung, Minjeong Kwon, Soonhee Lee. 84

86 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Physical Methods of Delivery I continued 490 Lipoplex Loaded Microbubbles for Gene Delivery: A Trojan Horse Controlled by Ultrasound Ine Lentacker, Stefaan C. De Smedt, Jo Demeester, Niek N. Sanders. 491 Local Delivery of Plasmid DNA into Skeletal Muscles by Echo-Contrast Gas Entrapping Liposomes, Bubble Liposomes Yoichi Negishi, Yoko Endo, Keiko Matsuo, Ryo Suzuki, Tomoko Takizawa, Kazuo Maruyama, Yukihiko Aramaki. 492 Comparison of the Sonoporation with the Cationic Polymer and Liposome by In-Vitro Gene Transfection Efficiencies and Toxicities Jeonghyun Park, Changshin Yoon, Hyesook Chung, Minjeong Kwon, Soonhee Lee. 493 Agitation during sirna-cationic Liposome (CL) Complex Formation Is a Key Step To Improve In Vitro RNA Interference by Lipofection Jose M. Barichello, Tatsuhiro Ishida, Luiz A. L. Soares, Shinji Kizuki, Tatsuaki Tagami, Kiyomi Hirose, Hideo Kobayashi, Kouichi Hashimoto, Hiroshi Kikuchi, Hiroshi Kiwada. 494 Towards Non-Viral Gene Therapy with Plasmid DNA Encapsulated in Pegylated Immuno-Liposomes (PILs) Torben Gjetting, Thomas L. Andresen, Thomas G. Jensen. 495 Thermosensitive Non-Viral Vectors: Combination of Gene Therapy with Hyperthermia Arkadi Zintchenko, Alenka Schwerdt, Manfred Ogris, Lars Lindner, Ernst Wagner, Massimo Concia. 496 The Novel Peptide Transporter System of p14 and p16 to Prostate Cancer Cells Satoshi Anai, Motoyoshi Tanaka, Nobumichi Tanaka, Kiyohide Fujimoto, Yoshihiko Hirao, Hirotsugu Uemura. 497 A Dose-Ranging Study of AAV2/1/SERCA2a Delivered Via the V-Focus Cardiac Delivery System in an Ovine Pacing Model of Heart Failure Melissa J. Byrne, Adam Bilney, Tuan Doan, John M. Power, David M. Kaye. 498 Hydrodynamics-Based Transfection Procedure Transiently Activates Expression of Preexistent Transgene Hiroshi Ochiai, Masahiro Fujimuro, Hideyoshi Yokosawa, Hideyoshi Harashima, Hiroyuki Kamiya. 499 Sequence Preferences of Plasmid-Microtubule Interactions Erin E. Vaughan, David A. Dean. 500 High Efficacy of High and Low Voltage Pulse Combinations for Gene Electrotransfer in Muscle, Liver, Tumor and Skin Franck M. André, Julie Gehl, Gregor Sersa, Véronique Préat, Natasa Pavsel, Jens Eriksen, Muriel Golzio, Maja Cemazar, Pernille H. Moller, Marie-Pierre Rols, Damijan Miklavcic, Eberhard Neumann, Justin Teissié, Lluis M. Mir. 501 Cyclic Stretch Following Electroporation Enhances Gene Transfer to the Lung Christopher D. Kaufman, David A. Dean. 502 An Endoscopic Tool for Targetted Gene and Drug Delivery Declan M. Soden, Mira Sadadcharam, John Piggott, Timothy Doody, Gerald C. O Sullivan. 503 A CE-Marked Automated Device for Electroporation- Enhanced DNA Delivery in Humans Rune Kjeken, Iacob Mathiesen, Feng Lin, Lei Zhang, Dietmar Rabussay, Torunn E. Tjelle. 504 A Single-Needle Device for Electroporation-Enhanced DNA Delivery Feng Lin, Kate Broderick, Jonas Söderholm, Kristen Smits, Torunn E. Tjelle, Dietmar Rabussay, Iacob Mathiesen, Rune Kjeken. Non-Viral Vectors: Intracellular Trafficking 505 Intracellular Trafficking of Oligonucleotides Delivered with Poly(Glycoamidoamine)s Katye M. Fichter, Theresa M. Reineke. 506 Analysis of the Intracellular Barriers Encountered by Nonviral Gene Carriers Following Localized Delivery to Axon Terminals Jamie M. Bergen, Suzie H. Pun. 507 Nuclear Accumulation of Plasmid DNA Can Be Enhanced by Non-Selective Gating of the Nuclear Pore Roosmarijn E. Vandenbroucke, Bart Lucas, Joseph Demeester, Stefaan C. De Smedt, Niek N. Sanders. FRIDAY, JUNE 1 85

87 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Non-Viral Vectors: Intracellular Trafficking continued 508 Intracellular Trafficking of DNA Nanoparticle and Nucleolin Antibody Xuguang Chen, Pamela B. Davis. 509 Fusogenic Segments of Glycoprotein H from Herpes Simplex Virus Enhance Transfection Efficiency of Cationic Liposomes Ying Tu, Ji-seon Kim. 510 An AlphaScreen Based Assay To Analyse Binding Properties of Engineered Histones with Application in Gene Therapy Kylie M. Wagstaff, David A. Jans. 511 Identification of Proteins Interacting with DNA Complex in Cytosol Takuro Niidome, Takayuki Iida, Takeshi Mori, Yoshiki Katayama. 512 Comparison of Two Nuclei Isolation Protocols for the Determination of the Number of Plasmid DNA Copies in the Nucleus after Transfection Marieke A. E. M. van der Aa, Richard N. Cohen, Ai Ping Lee, Francis C. Szoka, Jr. 513 Selective Penetration of Cancer Cells by Cupredoxins and Derivative Peptides as Potential Vectors for Gene Delivery Tohru Yamada, Brad N. Taylor, Mehta R. Rajeshwari, Fatima Lekmine, Konstantin Christov, Anne Shilkaitis, Laura Bratescu, Albert Green, Ananda M. Chakrabarty, Craig W. Beattie, Tapas K. Das Gupta. 514 Overcoming Hurdles of Nonviral Gene Delivery to Nonproliferating Cells by RNA Based Gene Transfer Shaomin Zou, James G. Hecker. 515 Novel Hybrid Vectors for Gene Delivery: Exploiting Viral Strategies with Non-Viral Vectors Vinod K. Valluripalli, Atan Rentsendorj, Hasmik Agadjanian, Jun Ma, Medina-Kauwe Lali. 516 Cdk2 Phosphorylation Correlates Positively with Cell Surface Nucleolin Expression Xuguang Chen, Pamela B. Davis. 517 DNA Nanoparticles Labeled with Tunable Nanocrystals: Demonstration of Coupling and Visualization by Fluorescence Microscopy Wenchao Sun, Anna C. S. Samia, Pamela B. Davis. 518 Nuclease Stability of sirna and DNA Condensed with PEGylated Polylysine Ozge Sesenoglu-Laird, Amanda G. Svenson, Olena Tyr, Tomasz H. Kowalczyk, Robert C. Moen, Mark J. Cooper. 519 DNA Delivery Was Enhanced by Novel Protein Transduction Domain, Hph-1 Eun S. Kim, Chang G. Lee, Seung W. Yang, Young C. Sung, Sang K. Lee. 520 Gene Transfection to Cells from Apical Side Takahito Kawano, Kousuke Yakumaru, Shuzo Yamashita, Takuro Niidome, Yoshiki Katayama. 521 Isolation of Human Fetal Myoblasts and Its Application for Microencapsulated Gene Therapy Anna A. Li, Jacqueline Bourgeois, L. Yoshida, Patricia Chang, Murray A. Potter. 522 An Inhibitor of the Fumarylacetoacetate Hydrolase (Fah) To Select for Gene Corrected Hepatocytes In Vivo Karsten Wursthorn, Nicole K. Paulk, John F. Witte, Raynard L. Bateman, Milton Finegold, Ronald W. McClard, Markus Grompe. 523 Peptide Modified Plasmid DNA for Gene Delivery Nicholas J. Baumhover, Mahesh Pavar, Garrett R. Rettig, Kevin G. Rice. 524 Non-Viral Gene Transfer Enhanced by a Nuclear Localizing Peptide Garrett R. Rettig, Kevin G Rice. 741 Chemical Modification of Oligonucleotides and DNA To Enable the Engineering of Improved Gene Therapy Vectors Oana M. Martin, Virginia M. Platt, Dipali Ruhela, Francis C. Szoka. Neurologic I 525 Evaluation of Formulation and Stability of Investigational Gene Transfer Vectors for Leber Congenital Amaurosis Olga Zelenaia, Xingge Lui, McDonnell Jennifer, Hauck Bernd, Al M. Maguire, Jean Bennett, Katherine A. High, J. Fraser Wright. 526 Antioxidant Gene Delivery To Mitigate Neurotoxicity in an Experimental Model of Chronic Brain Damage Due to HIV-1 Envelope Glycoprotein gp120 Jean-Pierre Louboutin, Lokesh Agrawal, Beverly A. S. Reyes, Elisabeth J. van Bockstaele, David S. Strayer. 86

88 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Neurologic I continued 527 Macrophage Delivery of GDNF Ameliorates Neurodegeneration in Parkinson s Disease Qing Zhou, Syed Z. Imam, Weijing He, Zhiqiang Liu, Robert A. Clark, James L. Roberts, Senlin Li. 528 Modulation of the Human Rhodopsin Expression by Artificial Zinc Finger Transcription Factors To Treat Dominant Retinal Degenerations Claudio Mussolino, Daniela Sanges, Valeria Marigo, Germana Meroni, Enrico M. Surace. 529 raav Mediated Parkin Over-Expression Partially Ameliorates the Behavioral Effects of the 6-Hydroxy Dopamine Lesion Fredric P. Manfredsson, Alfred S. Lewin, Corinna Burger, Nicholas Muzyczka, Ronald J. Mandel. 530 Protecting from Abnormal Blood Brain Barrier Permeability by Antioxidant Gene Delivery to the CNS Using SV40-Derived Vectors Jean-Pierre Louboutin, Lokesh Agrawal, Celestine Wanjalla, Beverly A. S. Reyes, Elisabeth J. van Bockstaele, David S. Strayer. 531 Cell Targeting in the CNS Using HIV-1-Based Lentiviral Vectors Bearing Alternative Glycoproteins and Promoters Thais Federici, Robert H. Kutner, Noel Tordo, Jun Yang, Erin Carlton, Nicholas M. Boulis, Jakob Reiser. 532 Trans-Synaptic Neurotoxicity of HIV-1 gp120 and Trans- Synaptic Protection Using rsv40 Vectors Delivery Antioxidant Enzymes Jean-Pierre Louboutin, Lokesh Agrawal, Beverly A. S. Reyes, Elisabeth J. van Bockstaele, David S. Strayer. 533 AAV-Mediated Aipl1 Gene Replacement in a Mouse Model of Leber Congenital Amaurosis Claudio Mussolino, Simona Neglia, Chiara Abrescia, Enrico M. Surace. 534 Improvement of Parkinson s Disease and Increase Action of Astrocytes by Human HGF Akihito Ito, Akihiko Ishida, Hiromi Koike, Toshikazu Nakamura, Ryuichi Morishita. 535 Axonal Regeneration of Retinal Ganglion Neurons Using Immortalized Adult Human Olfactory Ensheathing Glia M. Teresa Gallego-Hernández, M. Jesús Martín-Bermejo, Vega García- Escudero, Diana Simón, Ana García, Alberto Rábano, Javier Díaz-Nido, Jesús Ávila, Filip Lim, M. Teresa Moreno-Flores. 536 Activation of the JNK Pathway in a Rat Model of HD and Implication in the Pathology Valerie Perrin, Noelle Dufour, Cedric Raoul, Patrick Aebischer, Ruth Luthi-Carter, Nicole Deglon. 537 Human Studies as Basic Investigations: Patterns of Citation for Gene Transfer Phase I Clinical Trials in Malignant Glioma David L. McLauchlan, Jonathan Kimmelman. 538 IFN-γ Secretion Is Polarized at Effector Immunological Synapses (IS) In Vivo Carlos Barcia, Kurt M. Kroeger, Chunyan Liu, Kolja Wawrowsky, Maria G. Castro, Pedro R. Lowenstein. 539 Investigating the Role of MCP-1 in Early Alzheimer s Disease Via raav-mediated Hippocampal Gene Transfer in a Triple Transgenic Mouse Model Maya K. Desai, Michael A. Mastrangelo, Michelle C. Janelsins, Michael Valentino, Maria E. Frazer, Wade C. Narrow, Howard J. Federoff, William J. Bowers. 540 Gene Therapy Approach for Transthyretin Amyloidosis Using a Lentiviral Vector Expressing the Transsuppressor Variant Masakazu Kamata, Melisa T. Susanto, Irvin S. Y. Chen. 541 Induced Collateral Circulation by Hypoxia-Inducible Factor- 1α Decreased Cerebral Infarction in Rat Kazunori Shimotaka, Tatsuya Abe, Mitsuhiro Anan, Tohru Kamida, Keisuke Ishii, Minoru Fujiki, Hidenori Kobayashi. 542 Non-Viral Gene Therapy Technology in the Treatment of Chronic Pain Vanessa E. Gabrovsky, Jason M. Cuellar, Michele P. Calos, David C. Yeomans. 543 Role of Anti-Oxidant Enzymes Delivered Using Recombinant SV40-Derived Vectors in Protecting from Tat-Mediated Neurotoxicity In Vivo Lokesh Agrawal, Jean-Pierre Louboutin, Christina R. Maxwell, Beverly A. S. Reyes, Elisabeth Van Bockstaele, David S. Strayer. FRIDAY, JUNE 1 87

89 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Neurologic I continued 544 Constitutive GABA Expression Via a Recombinant Adeno- Associated Virus Consistently Attenuates Neuropathic Pain Boyoung Lee, Jaehyung Kim, Sung Jin Kim, Heuiran Lee, Jin Woo Chang. 545 Attenuation of Functional Deficits in the QA Model of Huntington s Disease Following AAV Therapeutic Delivery Adrian P. Kells, Rebecca A. Henry, Stephanie M. Hughes, Richard L. M. Faull, Bronwen Connor. Gene Therapy of Bone and Cartilage 546 A New Ex-Vivo Approach Using Autologous Dermal Fibroblasts Expressing hlmp3 Induces Bone Formation and Bone Healing in Animal Models Enrico Pola, Wanda Lattanzi, Claudio Parrilla, Annarita Fetoni, Leonardo Oggiano, Giandomenico Logroscino, Andrea Gambotto, Giovanni Pecorini, Roberto Pola, Paul D. Robbins. 547 Bone Regeneration at the Site of Osteoporotic Bone Fracture with Xenogeneic Skeletal Muscle Cells Producing BMP-2 Youngsuk Yi, Moon Jong Noh, Chae-Lyul Lim, Jong-Pil Hyun, Hyeon- Youl Lee, Kyoung Baek Choi, Kwan Hee Lee. 548 Mesenchymal Stem Cells Transduced Ex Vivo with BMP2 Reduce the Bone Consolidation Period in a Canine Model of Mandibular Bone Distraction Victor H. Cervantes-Kardasch, Yanko Castro-Govea, Rocio Ortiz-Lopez, Eduardo Alvarez-Lozano, Herminia Martinez-Rodriguez, Gerardo Padilla-Rivas, Viktor Romero-Diaz, Christian I. Silva-Platas, Jose L. Vazquez-Juarez, Augusto Rojas-Martinez. 549 Bone Morphogenetic Protein-2 Gene Therapy on Ovariectomized Mice Model Using Ex Vivo Modified Mesenchymal Stem Cells Sanjay Kumar, Tim R. Nagy, Selvarangan Ponnazhagan. 550 Combination of Baculovirus-Mediated BMP-2 Expression and Rotating-Shaft Bioreactor for Cartilage Tissue Engineering Huang Chi Chen, Li Yu Sung, Ching Kuang Chuang, Hsiao Ping Li, Yu Chen Hu. 551 Single Clone Selection of TGF-β1 Expressing Chondrocytes Using Retroviral Vector for the Treatment of Cartilage Defects: Its Stability, Efficacy and Safety Vivian Yip, Sally Hwang, Lillian Yun, Jaehyung Yoon, Youngsuk Yi, Moon Jong Noh, Kwan Hee Lee. 552 Active Immunization Against Macrophage Migration Inhibitory Factor Using a Novel DNA Vaccine Prevents Inflammation and Destruction in Murine Models of Arthritis Shin Onodera, Yoshikazu Koyama, Shigeki Oshima, Jun Nishihira. 553 Efficient In Vivo Non-Viral Gene Therapy Using Hydrodynamic Delivery of Folate-PEG-Chitosan-DNA Nanoparticles in an Adjuvant-Induced Arthritis (AIA) Rat Model Huijie Wang, CoVi Tran, Julio C. Fernandes. 554 Human Vascular Endothelial Growth Factor Gene Transference in an Unipedicle TRAM Flap by Electroporation To Prevent Necrosis Bianca C. G. Lisboa, Fernando C. Rezende, Heitor C. Gomes, Sang W. Han, A. F. Lucca, L. M. Ferreira. 555 Viral Mediated Transfer of the ELOVL4 Gene Can Result in High Level ELOVL4 Protein Expression in Human RPE Cells, Increasing the Major Anti-Inflammatory Retinal Fatty Acid DHA Sergey S. Seregin, Maria O. Tikhonenko, Julia V. Busik, Andrea Amalfitano. 556 Fetal AAV-Mediated Intra-Thoracic Gene Transfer and 3D Reconstruction of Bioluminescent Images of Infant Rhesus Monkeys Expressing Firefly Luciferase Alice F. Tarantal, C. Chang I. Lee. Protecting against Infectious Agents Using Gene Transfer 557 Survival of the Fittest: Challenging Lentiviral Vector- Transduced Cells with HIV-1 Replication Stephen E. Braun, Gang Qiu, Fay E. Wong, Michelle Connole, Joern E. Schmitz, John J. Rossi, Dorothee von Laer, R. Paul Johnson. 558 Chimeric Human and Rhesus TRIM5α Expressed Via Lentiviral Vectors Potently Restrict HIV-1 Infection In Vitro in CD34 Cell Derived Macrophages and In Vivo in SCID-hu Derived T Cells Joseph S. Anderson, Ramesh Akkina. 88

90 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Protecting against Infectious Agents Using Gene Transfer continued 559 Gene Silencing Using SV(siRNA-CCR3) and Delivery of SV(SFv-CCR3) in CNS Protects Brain Microglia from Infection of Primary HIV Isolates Lokesh Agrawal, Christina R. Maxwell, Charles R. Mackay, Paul J. Peters, Paul R. Clapham, David S. Strayer. 560 Effectiveness of Combinatorial Gene Therapy in Protecting PBMC from HIV-1 Infection Elena I. Marusich, David S. Strayer. 561 Simultaneous Expansion of CMV and EBV Specific T Cells Using Chimeric CMVpp65:EBNA3C Transgene Expressing Peripheral Blood Antigen Presenting Cells Xiuli Wang, Hunsar B. Meechoovet, Wen-Chung Chang, Michael C. Jensen. 562 Foamy Anti-HIV Vectors with MGMT Selection Cassettes To Evaluate AIDS Gene Therapy Strategies in the Macaque SHIV Model Grant D. Trobridge, Robert Wu, Mark S. Carter, Brian C. Beard, Shiu- Lok Hu, Dorothee von Laer, John Rossi, Hans-Peter Kiem. 563 In Vivo Derivation of HIV-1 Resistant Transgenic T Cells by Lentiviral Vector Transduction of a Triple Combination of Anti-HIV Genes Anti-CCR5 Ribozyme, Tat-Rev sirna and a TAR Decoy Joseph S. Anderson, Ming-Jie Li, Brent Palmer, Leila Remling, Shirley Li, Priscilla Yam, Jiing-Kuan Yee, John Rossi, John Zaia, Ramesh Akkina. 564 A Cationic Peptide Encoded by Influenza Virus A (H5N1) Can Operate as a Protein Carrier In Vitro and In Vivo Julia Steitz, Sean Alber, Simon C. Watkins, Andrea Gambotto. 565 Complex Adenovirus-Mediated Expression of C, PreM, E, and NS1 Proteins of West Nile Virus Induces Both Humoral and Cellular Immune Responses Jennifer Schepp-Berglind, Min Luo, Danher Wang, David H. Holman, Brian D. Hoel, Jason A. Wicker, Alan D. T. Barrett, John Dong. 566 Repeated Exposure to HIV-1 Selects for Highly HIV-Resistant Cells in Gene-Modified, but Not Naive, Lymphocytes Elena I. Marusich, David S. Strayer. 567 Effect of Lentivirus Vector-Based shrna on Chemotherapeutic Inhibition of HIV: Demonstration of Antiviral Synergy Shirley Li, Keith Meinking, Haiting Li, John A. Zaia, John Rossi. 568 Generation of a Hybrid SHIV Carrying HIV-1 Integrase in SIVmac239, as a Means To Test the Effectiveness of Anti- Integrase Gene Therapy Elena I. Marusich, Mahender Singh, David S. Strayer. 569 RNAi Targeted to Cellular Genes Involved in the HIV Life Cycle (Cyclins and Cyclin-Dependent Kinases) Results in Inhibition of HIV Replication Mayur C. Tamhane, Ramesh Akkina. 570 Multi-Targeting, Bifunctional Small Interfering RNAs Jane Zhang, Pal Saetrom, Ali Ehsani, Lars Aagard, John J. Rossi. 571 Downregulation of CCR5 and Inhibition of HIV-1 with RNAi, Ribozyme and Single Chain Fv Antibody Delivered by SV40- Derived Vectors To Protect CCR5-Expressing Primary Cells: Monocyte-Derived Macrophages and Microglial Cells from R5-Tropic HIV-1 Alena A. Chekmasova, David S. Strayer. 572 Complete Knockdown of CCR5 by Lentiviral Vector Expressed sirnas for HIV Gene Therapy Joseph S. Anderson, Ramesh Akkina. 573 In Vivo Derivation of CXCR4 sirna Transgenic Thymocytes in SCID-hu Mice and Inhibition of HIV-1 Infection by Lentiviral Vectors Joseph S. Anderson, Ramesh Akkina. 574 Development of Simian Adenovirus Vector Based and CD8 T Cell Directed Novel Genetic Vaccines for Dengue Virus Infection Guang-Ping Gao, ZhiYan Dai, Xun Sun, Joanita Figueredo, Thomas Verakis, Gang Li, James M. Wilson. 575 Development of Anti-HIV Gene Therapy Strategy Using Novel Endoribonuclease, MazF Hideto Chono, Hajime Matsumura, Kazuya Matsumoto, Masamitsu Shimada, Seung Shin Yu, Karim Lee, Sujeong Kim, Junichi Mineno, Sunyoung Kim, Keiji Terao, Masayori Inouye, Ikunoshin Kato. FRIDAY, JUNE 1 89

91 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Protecting against Infectious Agents Using Gene Transfer continued 576 Gene Delivery Targeting HIV-1 Integrase Does Not Select for Mutations in the Integrase Gene Marlene S. Strayer, Dawn G. Maier, Julien Landre, David S. Strayer. 577 Development of Potent Anti-HIV Chimeric Immune Receptors Wen Yang, Li Lu, Richard P. Junghans. 578 Optimal Condition for Lentivirus Infection and Effective Reduction of HIV-1 Infectivity by Membrane Bound C- Peptides Kwnag-soo Ahn, Seok Kee Chang, Dokeun Kim, Su Jin Seo, Hyun Song, Tae Gyun Kim, Sun Hee Kim, Min Joung Choi, Won Shin. 579 Investigating the Relationship between Duration of Antigen Expression and the Magnitude and Phenotype of the CD8+ T Cell Response Elicited by Recombinant Adenovirus Jonathan D. Finn, Tony Yang, Jamie Millar, Carole Evelegh, Robin J. Parks, Jonathan L. Bramson. 580 Zinc-Finger Nucleases Inactivating Hepatitis B Virus Genomic DNA Thomas J. Cradick, Andrew C. Jamieson, Anton P. McCaffrey. Cancer Immunotherapy II 581 Intracranial Glioma Tropism of Genetically Modified T Lymphocytes Following CNS Intraparenchymal Adoptive Transfer Reena P. Vishwanath, Christine E. Brown, Brenda Aguilar, Wen-Chung Chang, Michael C. Jensen. 582 Genetic Delivery of Avastin, an Anti-VEGF Monoclonal Antibody, Suppresses Human Tumor Growth In Vivo Masaki Watanabe, Julie L. Boyer, Neil R. Hackett, JianPing Qiu, Ronald G. Crystal. 583 Utilization of a Novel Luciferase Construct To Demonstrate Trafficking of Genetically Targeted CD19 Specific T Cells to Systemic Human Acute Lymphoblastic Leukemia in a Mouse Renier J. Brentjens, Elmer Santos, Raymond Yeh, Yan Nikhamin, Krista La Perle, Steve Larson, Michel Sadelain. 584 Surface Expression of Anti-CD3 Single-Chain Antibody and CD86 on B16 Melanoma Cells Elicits Effective Anti-Tumor Immunity Bing-Mae Chen, Chen-Hsien Lee, Yi-Shen Chiang, Shih-En Chang, Steve R. Roffler. 585 In Vivo Activation of Endogenous, Pigment Epithelial Derived Factor (PEDF) Expression by a Zinc Finger Transcription Factor in Human Lung Cancer Cells Gary K. Lee, Yu-An Zhang, Nhu Tran, Andreas Reik, Steve H. Zhang, Philip D. Gregory, Dale Ando, John Nemunaitis, Alex W. Tong. 586 Investigation of Single Chain MHC Molecules for Specific Cancer Treatment Steve R. Roffler, Ya-Chen Li, Chia-Ming Chang, Mi-Hua Tao. 587 Redirecting Specificity of Lymphocytes to CD19+ Malignancies Using Non-Integrating Gene Transfer Carrie H. Yuen, Helen Andersson, Simon Olivare, Laurence J. Cooper. 588 Anticancer Activity of Oncolytic Adenovirus Vector Armed with IFN-alpha and ADP Is Enhanced by Pharmacologically- Controlled Expression of TRAIL Elena V. Shashkova, Mohan N. Kuppuswamy, William S. M. Wold, Konstantin Doronin. 589 Generation of a New Type of Yeast-Secreted Biotinylated Recombinant Antibodies Directed Against Mesothelin and Able To Prevent CA125/Mesothelin-Dependent Cell Attachment Nathalie Scholler, Barbara Garvik, Lindsay Bergan, Barry Nevin, Jennifer Gross, Nicole Urban. 590 Antitumor Effect of Intravenous Administration of Oncolytic Herpes Simplex Virus Expressing Interleukin 12 on Systemic Metastases Yi Guan, Yasushi Ino, Fukuhara Hiroshi, Tomoki Todo. 591 Combination Treatment Strategies That Modify Ceramide Metabolism To Increase the Efficacy of FasL Gene Therapy Lorianne S. Turner, Xiang Lu, Saeed Elojeimy, Ayman E. M. Mahdy, Antonio F. Saad, Jian-Yun Dong, Terrence Day, Thomas Keane, Yusuf Hannun, James S. Norris. 592 In Vivo Monitoring of Efficacy of Human Genetically Modified PSMA-Specific Lymphocytes Konstantin Dobrenkov, Malgorzata Olszewska, Yuri Likar, Jelena Vider, Larissa Shenker, Michel Sadelain, Vladimir Ponomarev. 90

92 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Cancer Immunotherapy II continued 593 Enhanced T Cell-Mediated Antitumor Efficacy of Oncolytic Adenovirus Expressing IL-12 and GM-CSF Kyung-Ju Choi, Hye-Ran Park, Il-Kyu Choi, Song-Nam Zhang, Hye-Jin Choi, Chae-Ok Yun, Joo-Hang Kim. 594 The CE7R Chimeric Antigen Receptor Re-Directs T Cell Specificity to a Broad Range of L1-CAM Expressing Human Malignancies Michael Stastny, Christine E. Brown, Wen-Chung Chang, Michael C. Jensen. 595 Activation of Tumor-Specific CD8 + T Cells after Intratumoral Ad5-TRAIL/CpG ODN Combination Therapy Rebecca L. VanOosten, Thomas S. Griffith. 596 Imaging of HSV1-tk Expressing Cytolytic T Cell Clones in Glioma Patients with [ 18 F]FHBG Positron Emission Tomography Shahriar S. Yaghoubi, Michael C. Jensen, Yingbing Wang, Nagichettiar Satyamurthy, Mohammad Namavari, Frederick T. Chin, Murugessan Subbarayan, Czernin Johannes, Sanjiv S. Gambhir. 597 Expression of Biologically Active Anti-HER2 Antibody from an AAV Gene Transfer Vector Guoqing Wang, JianPing Qiu, Julie L. Boyer, Neil R. Hackett, Ronald G. Crystal. 598 Targeted Elimination of Stem-Like Glioma Progenitors with Genetically Engineered Cytolytic T Cells Christine E. Brown, Renate Starr, Brenda Aguilar, Michael C. Jensen. 599 A Critical Role for Receptor Dimerisation and Transmembrane Domains for the Optimal Function of Chimeric Immune Receptors John S. Bridgeman, Robert E. Hawkins, David E. Gilham. Cancer: Targeted Therapy II 600 Valproic Acid (VPA) Enhances the Antitumor Effect of Telomerase-Specific Replication-Competent Adenoviral Agent OBP-301 (Telomelysin) in Human Lung Cancer Cells Yuichi Watanabe, Yuuri Hashimoto, Shunsuke Kagawa, Hitoshi Kawamura, Katsuyuki Nagai, Noriaki Tanaka, Yasuo Urata, Toshiyoshi Fujiwara. 601 Switching Adenovirus Fibers for Retaining Gene Delivery Efficacy in the Presence of Neutralizing Antibodies Merja Särkioja, Sari Pesonen, Tanja Hakkarainen, Jarmo Salo, Anna Kanerva, Akseli Hemminki. 602 Combination Therapy with Radiation or Cisplatin Enhances the Potency of Ad5/35 Chimeric Oncolytic Adenovirus in a Preclinical Model of Head and Neck Cancer Shanthi Ganesh, Melissa Gonzalez, Douglas Gibbons, Melinda VanRoey, Karin Jooss. 603 Chemoprotection of Human Hematopoietic Stem Cells by Lentiviral Transfer of Multidrug-Resistance 1 Gene in Combination with the O 6 -Methylguanine-DNA Methyltransferase P140K Mutant Gene Isabel Spier, Patrick Maier, Stephanie Laufs, Frederik Wenz, Stefan Fruehauf, Jens W. Zeller. 604 A Dual-Action, Armed Replicating Adenovirus for the Treatment of Bone Metastases of Breast Cancer James J. Cody, Angel A. Rivera, Sherry W. Yang, Gray R. Lyons, Minghui Wang, Gene P. Siegal, Joanne T. Douglas. 605 Compassionate Approval Process for Experimental Gene Based Products Gladice Wallraven, John J. Nemunaitis, Phillip B. Maples. 606 Delivery of the CD40 Ligand (CD40L, CD154) Transgene by an Oncolytic Adenovirus Construct: A Targeted Therapeutic Approach for Human Multiple Myeloma Alex W. Tong, Margret S. Rodrigues, Erica M. Gomes, Reuben Hernandez, Joseph Newman, John J. Nemunaitis, Marvin J. Stone. 607 The Application of Tumor Stem Cells for Adenoviral-Based Glioma Gene Therapy Ilya V. Ulasov, Matthew A. Tyler, Adam M. Sonabend, Maciej S. Lesniak. FRIDAY, JUNE 1 91

93 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Cancer: Targeted Therapy II continued 608 Expression of Tissue Inhibitor of Matrix Metalloproteinases (TIMP)-3 by an Oncolytic HSV Enhances Antitumor Efficacy Against Neural Tumors and Tumorspheres Yonatan Y. Mahller, Sachin S. Vaikunth, William H. Baird, Maria C. Ripberger, Jose A. Cancelas, Yoshinaga Saeki, Timothy M. Crombleholme, Timothy P. Cripe. 609 Kringle 1 Domain of Human Hepatocyte Growth Factor (HGFK1) Inhibits the Growth and Metastasis of Hepatocellular Carcinoma in Rats Zan Shen, Zhen Fan Yang, Yi Gao, Ronnie T. P. Poon, Sheung Tat Fan, Ming-liang He, Tsai-ping Li, Ren-bao Gan, Hsiang-fu Kung, Marie C. Lin. 610 Treatment of Metastatic Colorectal Cancer with Triple Capsid-Modified Adenoviruses Featuring Reduced Liver Tropism Sergio Lavilla-Alonso, Gerd J. Bauerschmitz, Ramon Alemany, Neus Bayo-Puxan, Anna Kanerva, Kilian Guse, Akseli Hemminki. 611 Development of a Selective Non-Viral Gene Directed Enzyme/Pro-Drug Cancer Therapy Based on a Chimeric Reactive Oxygen Species-Response Element Lucía L. Policastro, Irene L. Ibañez, Hebe A. Duran, Osvaldo L. Podhajcer. 612 Assessment for Antitumor Activity of Telomerase-Specific Oncolytic Virotherapy in the Hypoxic Microenvironment Yuuri Hashimoto, Teraishi Fuminori, Yuichi Watanabe, Ryo Sakai, Shinji Kuroda, Shunsuke Kagawa, Yasuo Urata, Noriaki Tanaka, Toshiyoshi Fujiwara. 613 Tight Regulation of Ionizing-Radiation Inducible Transgene Expression by Lentivirus Vectors Christian E. Badr, Bakhos A. Tannous. 614 Proteomics Analysis in the Development of Molecular Precision Approaches for Cancer Therapy Yuqiao Shen, Phillip B. Maples, Padmasini Kumar, Donald Rao, Alex W. Tong, Neil Senzer, John J. Nemunaitis. 615 Short Hairpin RNA-Expressing Oncolytic Adenovirus- Mediated Inhibition of IL-8: Effects on Anti-Angiogenesis and Tumor Growth Inhibition Chang Xiu Lin, Ji-Young Yoo, Jae-Sung Kim, Sang-Joon Sin, Joo-Hang Kim, Chae-Ok Yun. 616 Salmonella choleraesuis as an Anticancer Agent in a Syngeneic Model of Orthotopic Hepatocellular Carcinoma Che-Hsin Lee, Chao-Liang Wu, Ai-Li Shiau. 617 A Serotype 3 Retargeted Oncolytic Adenovirus That Expresses a TK-GFP Fusion Protein for Imageable Combined Modality Treatment of Ovarian Cancer Mari Raki, Anna Kanerva, Tanja Hakkarainen, Gerd Bauerschmitz, Merja Sarkioja, Renee A. Desmond, Akseli Hemminki. 618 A Novel Chromogranin-A Promoter-Driven Oncolytic Adenovirus for Midgut Carcinoid Therapy Justyna Leja, Helena Dzojic, Elisabet Gustafson, Kjell Oberg, Valeria Giandomenico, Magnus Essand. 619 Ad5/19p for Targeting of Renal Cancer and Detargeting the Liver Iulia Diaconu, Laura Denby, Gerd Bauerschmitz, Kilian Guse, Anna Kanerva, Joao D. Dias, Andrew H. Baker, Akseki Hemminki. 620 An Efficient and Safe Herpes Simplex Virus Type 1 Amplicon Vector for Transcriptionally Targeted Therapy of Human Hepatocellular Carcinomas Kian Chuan Sia, Lv Miao, Grace Y. Wang, Ivy A. W. Ho, H. Huynh, Kam M. Hui, Paula Y. P. Lam. 621 Fluorescently Tagged Conditionally Replicative Adenovirus Via Modification with Protein IX Red Fluorescent Protein Fusion Ana Nedeljkovic-Kurepa, Heike Creutz, Mariam A. Stoff-Khalili, Yoshinobu Odaka, Jagat Poduturri, Xiao L. Li, Angel A. Rivera, David T. Curiel, J. Michael Mathis. 622 Fiber Region-Modified Adenoviruses Produced Anti-Tumor Effects to CAR-Low Expressing Tumors Masatoshi Tagawa, Kiyoko Kawamura, Guangyu Ma, Quanhai Li, Osamu Shimozato, Ayako Sato, Taketo Suzuki, Nobuo Suzuki, Hideaki Shimada, Takenori Ochiai. 92

94 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Cancer: Targeted Therapy II continued 623 Dual Luciferase Imaging for Evaluation of the Specificity of Adenoviruses with Hypoxia Response Elements in Renal Tumors Kilian Guse, Tanja Hakkarainen, Minna Eriksson, Anna Kanerva, Akseli Hemminki. 624 Adenovirus Delivered to Tumor Endothelium by Angio- Adenobodies Haisma J. Haisma, Marieke Geel, Anu Kariath, Anna Rita Bellu. Cancer: Targeted Therapy III 625 Inhibition of Hepatic Metastasis of Colon Carcinoma Cells by RNA Interference-Mediated Suppression of HIF-1α in Tumor Cells and Hepatocytes Yuki Takahashi, Makiya Nishikawa, Yoshinobu Takakura. 626 Suppression of Renal Cell Carcinoma Growth and Metastasis with Adenoviral Delivery of Chimeric Antiangiogenic Proteins Thomas A. Gardner, Matthew J. Mellon, Miwon Ahn, Yan-Ping Zhang, Juan A. Jimenez, Chinghai Kao. 627 Development of a Highly Efficient Targeted Gene Therapy Vector for the Treatment and Diagnosis of Metastatic Breast Cancer Steven T. Huyn, Makoto Sato, Mai Johnson, Jeremy Burton, Lily Wu. 628 Prevention of Irradiation-Induced Salivary Hypofunction by Microvessel Protection in Mouse Salivary Glands Ana P. Cotrim, Anastasia L. Sowers, James B. Mitchell, Bruce J. Baum. 629 An Adenovirus Vector Re-Targeted to Her2/neu by an Affibody TM Molecule and De-Targeted from CAR, a v Integrin and HSG Binding: In Vitro and In Vivo Data Maria K. Magnusson, Petra Henning, Saw See Hong, Nagy A. Habib, Stefan Ståhl, Pierre Boulanger, Leif Lindholm. 630 VEGFR1 Targeted Oncolytic Virotherapy of Breast Cancer Lyudmila N. Kaliberova, Valentina Krendelchtchikova, Donald J. Buchsbaum, Sergey A. Kaliberov. 631 Therapeutic Efficacy of Midkine Promoter-Based Conditionally Replicative Adenovirus Vector for Targeting the Midkine-Expressing Human Bladder Cancer Model Shuji Terao, Toshiro Shirakawa, Shuji Kubo, Katsuyuki Hamada, Masatoshi Tagawa, Atsushi Takenaka, Masato Fujisawa, Akinobu Gotoh. 632 Improving Cancer Gene Therapy for Advanced Head and Neck Cancer with a Multi-Modal Therapy Approach Joao D. Dias, Minna Eriksson, Dung-Tsa Chen, Kilian Guse, Mikko Tenhunen, Reidar Grenman, Mikko Savontaus, Akseli Hemminki. 633 Lentiviral-Mediated Gene Delivery of IFN-beta Strongly Inhibits Pancreatic Cancer-Derived Cells Proliferation Emmanuel Ravet, Fabian Gross, Louis Casteilla, Lucien Pradayrol, Louis Buscail, Pierre Cordelier. 634 MDR1 Gene Transfer Using a Lentiviral SIN Vector Confers Radioprotection to Human CD34+ Haematopoietic Progenitor Cells Patrick Maier, Carsten Herskind, Katharina Fleckenstein, Stephanie Laufs, Jens W. Zeller, Stefan Fruehauf, Frederik Wenz. 635 Conditionally Replicating Transductionally Targeted Adenovirus Ad5/35-TERT Is More Effective Against Head and Neck Cancer Than Non-Targeted CRAD Ad5-TERT Raine Toivonen, Elina Suominen, Reidar Grenman, Mikko Savontaus. 636 Development of Prostate-Specific Conditional Replication- Competent Adenovirus for Prostate Cancer Gene Therapy Yi Lu, Guimin Chang, Jun Zhang. 637 Targeted Cancer Gene Therapy Using AAV2-Mediated Anti- Angiogenesis Approaches Keerang Park, Young-Hwa Cho, Wun-Jae Kim, Wongi Seol, Eui-Sic Cho, Yeun-Ju Kim, Sung-Ha Cho, Hee-Jong Kim, Bong-Su Kang, Won-Jin Ji. 638 Telomerase-Targeted Oncolytic Adenovirus in Combination with Chemotherapy Elicits Enhanced Cytopathic and Anti- Tumor Effects in Head & Neck Squamous Cell Carcinoma Pyung-Hwan Kim, JinSun Kim, Min Jung Kim, O.-Jun Kwon, Joo-Hyuk Sohn, Chae-Ok Yun, Joo-hang Kim. 639 Irradiation Prior to Adenovirus Treatment Increases Transgene Expression in Prostate and Breast Cancer Cells Petri Nokisalmi, Maria Rajecki, Tanja Hakkarainen, Mikko Tenhunen, Akseli Hemminki. FRIDAY, JUNE 1 93

95 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Cancer: Targeted Therapy III continued 640 Evaluation of Biodistribution and Safety of DWP418, Relaxin Expressing Oncolytic Adenovirus Therapeutics Kyunghyun Min, Jaemook Choi, Kinam Kim, Joohang Kim, Chaeok Yun, Yoewook Koh, Hyunsoo Kim. 641 Triple Helix-Forming Oligonucleotides Specifically Reduces ATM Transcription, Translation, and Radiosensitivity in Human Glioma Cells Chu-Chiao Wu, Chi-Shiun Chiang. 642 Regio-Specific Mutagenesis of Thermostabilized Yeast Cytosine Deaminase for Improved 5-Fluorocytosine Activity Marilyn Sanchez-Bonilla, Candice Willmon, Margaret E. Black. 643 Development of Tenascin-C Binding Peptide Aptamers as the Tumor Targeting Tools Mee Young Kim, Ji Sun Yang, Heuiran Lee, Keerang Park, Sunjoo Jeong. 644 Whither Therapeutic Targeting? The Sorcerer s Apprentice Finally Meets Jasper Johns* Neil Senzer, John J. Nemunaitis. Hematologic II 645 Friend Murine Erythroleukemia (MEL) Cells Do Not Reliably Predict Levels of Globin Transgene Expression In Vivo Leszek Lisowski, Eirini Papapetrou, Michel Sadelain. 646 Restoration of von Willebrand Factor Function in a Murine Model of Severe von Willebrand Disease after Liver Specific Gene Transfer Simon F. De Meyer, Marinee K. Chuah, Thierry VandenDriessche, Nele Vandeputte, Inge Pareyn, Inge Petrus, Hans Deckmyn, Karen Vanhoorelbeke. 647 Gene Therapy of MPL Deficiency: A Paradigm for Gene Addition Therapy of Growth Factor Receptors Daniel Wicke, Ute Modlich, Johann Meyer, Matthias Ballmaier, Cornelia Rudolph, Brigitte Schlegelberger, Karl H. Welte, Christopher Baum. 648 Pre-Clinical Evaluations of the Biosafety of a Lentiviral Vector Developed for the Gene Therapy of Wiskott Aldrich Syndrome Anne Galy, Sabine Charrier, Julie Mantovani, Javier Perea, Maria- Antonietta Zanta-Boussif, Marie Liabeuf, Marie Montus, Adrian Thrasher, Francesco Marangoni, Samantha Scaramuzza, Anna Villa, Alessandro Aiuti, Loïc Dupré, Maria-Grazia Roncarolo. 649 Generation of Efficient Human Blood Progenitor-Targeted Recombinant Adeno-Associated Viral Vectors by Applying an AAV Random Peptide Library on Primary Human Hematopoietic Progenitor Cells Leopold Sellner, Marius Stiefelhagen, Juergen A. Kleinschmidt, Frederik Wenz, Stefan Fruehauf, W. Jens Zeller, Marlon R. Veldwijk. 650 In Vivo Selection of Hematopoietic Cells Following Retroviral Gene Transfer in a Dog with Mucopolysaccharidosis VII Thomas M. O Malley, Xiucui Ma, Uimook Choi, Patricia O Donnell, Ping Wang, Douglas R. Kennedy, James Rhodes, Caroline Bryan, Peter J. Felsburg, Harry L. Malech, Katherine P. Ponder, Mark E. Haskins. 651 Gene Therapy for Beta-Thalassemia: Studies on Human Cells Emanuela A. Roselli, Rossano Cesari, Annarita Miccio, Francesca Tiboni, Paola Corbella, Claudia Rossi, Erika Biral, Sarah Marktel, Michael Antoniou, Marco Andreani, Guido Lucarelli, Giuliana Ferrari. 652 Cytidine Deaminase Gene Transfer Allows Transient In Vivo Enrichment of Transduced Hematopoiesis by Extended Low Dose Cytarabine Treatment Ina Rattmann, Jeff Bailey, Victoria Summey, Jamie Siefring, David A. Williams, Thomas Moritz. 653 Efficient Transduction of Thalassemic CD34+ Cells by Novel γ-globin Gene Lentiviral Insulated Vectors with the HPFH-2 Enhancer and the -117 HPFH Promoter Mutation Eleni Papanikolaou, Maria Georgomanoli, Nicholas P. Anagnou. 654 Construction of Foamy Virus Vectors for the Genetic Correction of beta-thalassemia John Morianos, Elena K. Siapati, George Vassilopoulos. 655 Segmental Trans-Splicing of the C57Bl/6 Murine von Willebrand Factor Pre-mRNA To Facilitate Packaging into Gene Transfer Vectors Fang Liu, Julie L. Boyer, Neil R. Hackett, Bishnu P. De, Ronald G. Crystal. 94

96 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Hematologic II continued 656 Lentiviral Vector-Mediated Correction of Artemis Deficient SCID and Functional Protein Analysis by FRET Megan M. Multhaup, Jennifer L. Gori, Andrea Karlen, Debra Swanson, Nikunj V. Somia, Morton J. Cowan, R. Scott McIvor. 657 Construction of a High Performance Lentiviral cdna Library Derived from Human Fetal Liver Ryo Kurita, Tatsuo Oikawa, Michiyo Okada, Tomoko Yokoo, Norio Komatsu, Yoshikuni Tanioka, Erika Sasaki, Kenzaburo Tani. 658 The Erythrocyte Immunity Research on Chronic Venous Insufficiency of Lower Limb Zhang Lan, Zhang Baigen. Immune Responses to Adenoviral Vectors 659 TLR4 and TRIF Play Positive and Negative Roles in Ad Induced Innate Immunity In Vivo Daniel M. Appledorn, Zachary Hartman, Jeannine M. Scott, Andrea Amalfitano. 660 Adenovirus Induced Innate Immune Responses Are Mediated in Part by the Presence of Natural Antibodies in Ad Naïve Mice Jeannine M. Scott, Tyler T. Voss, Daniel M. Appledorn, Andrea Amalfitano. 661 Dose-Limiting Toxicities Following Systemic Administration of Non-Human Primate Derived Adenoviral Vectors to Cynomolgus Macaques Suryanarayan Somanathan, Guang-Ping Gao, Martin Lock, Peter Bell, Rebecca Grant, Soumitra Roy, Arbansjust Sandhu, James M. Wilson. 662 Study of Adenovirus Vector-Induced Activation of Muscle Dendritic Cells and Analysis of Mouse Dendritic Cell Migration from the Tibialis Anterior Muscle Lei Wang, Paula R. Clemens. 663 Methotrexate Selection of Gene Modified T Cells with an Engineered Human Dihydrofolate Reductase (DHFR) Christine E. Brown, Wen-Chung Chang, Christine Wright, Araceli Naranjo, Jamie Wagner, Hunsar B. Meechoovet, Julie R. Ostberg, Michael C. Jensen. 664 Identification of TLR2 as a Mediator of the Innate Immune Response to Adenoviral Vectors Daniel M. Appledorn, Jeannine M. Scott, Andrea Amalfitano. 665 Host Response to Adenovirus, Helper-Dependent Adenovirus and Adeno-Associated Virus in Mouse Liver Paul Fawcett, Hiroyuki Nakai, Anja Ehrhardt, Mark A. Kay, Anton McCaffrey. 666 Effect of Anti-Adenoviral Immunity on Long-Term Vector and Islet Graft Function Jun Cheng, Jianmin Sun, Randall S. Sung. 667 Periocular Triamcinolone Enhances Intraocular Gene Expression Following Delivery by Adenovirus Marisol D. Cano, Choul Y. Park, Roy S. Chuck, Margaret Yew, Viet Nguyen, Jack Parker, Keisuke Mori, Peter L. Gehlbach. 668 Effect of Pre-Existing Anti-Adenovirus Immunity and Anti- Tumor Immunity on the Efficacy of Adenovirus Vectors in Immunocompetent Syrian Hamster Model Debanjan Dhar, Jacqueline F. Spencer, Karoly Toth, William S. M. Wold. 669 Involvement of MyD88 and TLR9 in the Innate Immune Response Elicited by Replication-Incompetent Adenovirus Vectors Tomoko Yamaguchi, Kenji Kawabata, Naoya Koizumi, Fuminori Sakurai, Kazuko Nakashima, Tomomi Sasaki, Naoki Okada, Hiroyuki Mizuguchi. 670 Administration of a Combination of a Nondepleting CD4 Antibody and Cyclosporine Is Efficient at Attenuating Humoral but Not Cellular Immunity after Adenovirus Serotype 5 Mediated Gene Transfer Jenny H. McIntosh, Melanie Cochrane, Andrew M. Davidoff, Amit C. Nathwani. Gene Expression in Animal Model Systems 671 Directing a Transgenic Erythropoietin Fusion Protein into the Regulated Secretory Pathway in Murine and Rat Salivary Gland Yuval Samuni, Ana P. Cotrim, Changyu Zheng, Gabor Z. Racz, Niamh X. Cawley, Peng Y. Loh, Bruce J. Baum. 672 Efficient Conversion of Embryonic Stem Cells into Myogenic Lineage Using an Inducible Gene Expression System Shigemi Kimura, Shiro Ozasa, Kaori Ito, Makoto Matsukura, Teruhisha Miike. FRIDAY, JUNE 1 95

97 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Gene Expression in Animal Model Systems continued 673 The Evaluation of Novel Photoreceptor Specific Promoters in an EIAV Lentiviral Vector for Targeted Gene Expression in the Photoreceptors of the Eye On Kan, Marjorie Nicoud, Sharifah Iqball, Stuart Sims, Jian Kong, Peter Gouras, Rando Allikmets, Stuart Naylor, Katie Binley. 674 Hypoxia-Regulated and Cell-Specific AAV Vectors Targeting the Retina and Retinal Pigmented Epithelium Christopher J. Dougherty, George-Wesley T. Smith, Kathleen Dorey, Keith A. Webster, Howard M. Prentice, Janet C. Blanks. 675 Enhanced Factor VIII Secretion in Hemophilia A Mice Using a Feline Immunodeficiency Virus Vector and Transgenic Modifications C. P. Chikkanna-Gowda, Litao Xie, Melissa A. Hickey, Patrick L. Sinn, Steven W. Pipe, Paul B. McCray, Jr. 676 Direct Comparison of Hepatocyte-Specific Expression Cassettes after Adenoviral Transfer and Hydrodynamic Transfer of Plasmids and Minicircles in C57BL/6 Mice Frank Jacobs, Jan Snoeys, Yingmei Feng, Eline Van Craeyveld, Désiré Collen, Donna Armentano, Seng H. Cheng, Bart De Geest. 677 AAV-Mediated IGF-1 Delivery to the Cervical Spinal Cord of the SOD1 Rat Model of ALS Via Intraparenchymal Injection Jun Yang, Erin Carlton, Thais Federici, Kathie M. Bishop, Carey Backus, Sang Su Oh, Qingshan Teng, Jonathon Riley, Mehdi Gasmi, Raymond T. Bartus, Eva L. Feldman, Nicholas M. Boulis. 678 Cell Permeable, Anti-Inflammatory Peptides Targeting NF-κB and NFAT Pathways Prevent the Onset of Diabetes in NOD Mice Khaja K. Rehman, Paul D. Robbins. 679 Sensitive and Precise Regulation of Haemoglobin after Gene Transfer of Erythropoietin to Muscle Tissue Using Electroporation Pernille H. Moller, Hanne Gissel, Julie Gehl. 680 Alcohol Drinker Rats Lower Their Ethanol Intake for One Month after a Single Injection of an Adenoviral Vector Carrying an Antisense Gene for Aldehyde Dehydrogenase Paula Ocaranza, María Elena Quintanilla, Lutske Tampier, Eduardo Karahanian, Amalia Sapag, Yedy Israel. 681 Improvement of Survival of Skin Flaps by Combined Gene Transfer of Hepatocyte Growth Factor and Prostacyclin Synthase Aya Nakagawa, Motokuni Aoki, Takashi Miyake, Suguru Shiraya, Toshikazu Nakamura, Toshio Ogihara, Yoshihiro Kimata, Ryuichi Morishita. 682 A New Experimental Model for HIV-1 Lesions in the Brain Based on SV40 Vectors Expressing Envelope Glycoprotein gp120 Jean-Pierre Louboutin, Lokesh Agrawal, Beverly A. S. Reyes, Elisabeth J. van Bockstaele, David S. Strayer. 683 Regulated Gene Expression in Human Hematopoietic Stem Cells after Transduction with Helper-Dependent Ad5/35 Vector Hongjie Wang, Andre Lieber. 684 Expression of Foxp3 in a Subset of Rat Dendritic Cells (DCs), Plasmacytoid DC, Generated from a Long-Term Culture of Bone Marrow Cells (BMC) Driven by FLt3-Ligand and IL-6 Ryota Haba, Naoko Fuji-Funeshima, Atsushi Tsuji, Xiao-Kang Li, Iwao Nozawa, Hiromitsu Kimura. 685 The Role of PTEN Tumor Suppressor Gene in Prostate Cancer Derived by Prostate Specific Deletion of PTEN Motoyoshi Tanaka, Atsushi Tomioka, Satoshi Anai, Marco Velasco, Hirotsugu Uemura. 686 Establishment of a Murine Colorectal Tumor Model for In Vivo Imaging and Development of Gene Therapies for Metastatic Disease Alice H. Hsu, Brent Soresen, Andrew C. Wilber, Daniel A. Saltzman, R.Scott McIvor, Lalitha R. Belur. 687 A Novel Patient-Specific Cancer Vaccine Approach Based on Genetically Altered Tumor Immune Profile and Xenograft Tumor Expansion Padmasini Kumar, Chris Jay, Ila Oxendine, Viviana Mangual, John J. Nemunaitis, Phillip B. Maples. 688 Aiming at Alcoholism with Ribozyme Genes: Silencing the mrna for an Alcohol Detoxifying Enzyme Lorena Lobos-González, Carlos Muñoz-Brauning, Amalia Sapag. 96

98 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Gene Expression in Animal Model Systems continued 689 Targeting Activator Protein-1 To Investigate Protective Phytocompounds Against UVB Radiation-Induced Photoaging in Mouse Vanisree Staniforth, Wen-Ching Huang, Ning-Sun Yang. 690 Corneal Gene Therapy: Past Experiences, Current Trends and Future Perspectives Eytan A. Klausner, Dan Peer, Robert L. Chapman, Richard F. Multack, Shridhar V. Andurkar. 691 Developmental Stage Determines Distribution of Organ Transduction after Extracoelomic Cavity Injection of Lentiviral Vector Masayuki Endo, Tiago H. Coelho, Phillip W. Zoltick, Antoneta Radu, Nidal Muvarak, Alan W. Flake. 692 Regulatory Elements for Improved Gene Therapy Vectors Amy C. Groth, George Stamatoyannopoulos, David W. Emery. Oligonucleotide Therapies I 693 Adverse shrna Cytotoxicity Can Accelerate Tumorigenesis in Genetically Predisposed Mice Dirk Grimm, Shelly Beer, Kim Komatsubara, Joyce S. Lee, Shan Koh, Lora Wang, Theresa A. Storm, Corrine R. Davis, Mark A. Kay, Dean Felsher. 694 RNA Interference Mediated by Cardiotropic AAV Vectors To Inhibit Coxsackievirus B3 Infections in Cardiac Cells Sandra Pinkert, Isaac Sipo, Lennart Suckau, Xiaomin Wang, Jens Kurreck, Heinz Zeichhardt, Wolfgang Poller, Henry Fechner. 695 In Vitro and In Vivo Evaluation of shrnas and mirna Shuttles for Therapeutic RNAi Ryan L. Boudreau, Alex Mas, Scott Q. Harper, Beverly L. Davidson. 696 Efficient Knock-Down of Prion Protein by Lentivectors Expressing Short Hairpin RNAs Andreas Hofmann, Sabina Eigenbrod, Saba Al-Khadra, Markus Moser, Uwe Bertsch, Hans Kretzschmar, Alexander Pfeifer. 697 Therapeutic Gene Knockdown with Wrapped Liposome- Mediated Systemic sirna Delivery Nobuhiro Yagi, Ichiro Manabe, Hidenori Takahashi, Jongheon Kim, Katsuhito Fujiu, Yumiko Oishi, Masahiro Yamauchi, Noboru Aoki, Yasuhiro Tamaki, Ryozo Nagai. 698 Mutation-Independent RNAi for Rhodopsin-Linked Autosomal Dominant Retinitis Pigmentosa Naomi Chadderton, Arpad Palfi, Mary O Reilly, Sophia Millington- Ward, Marius Ader, Gearoid Tuohy, Peter Humphries, Paul F. Kenna, G. Jane Farrar. 699 Allele-Specific Silencing of Mutant Huntingtin for Huntington s Disease Therapy Alex Mas-Monteys, Scott Q. Harper, Brian L. Gilmore, Patrick Staber, Chris Schaffer, Barry Polisky, Chandra Vargesse, Beverly L. Davidson. 700 Development of RNA Aptamers for Human Toll-Like Receptor 2 Hyoung-Rea Yoo, Hun-Young Kong, Han-Na Lee, Young-Ju Lee, Seong- Wook Lee, Jonghoe Byun. 701 Restoration of Dystrophin Expression in the Diaphragm of the Dystrophic mdx Mice by Polymer-Tagged Morpholino Antisense Oligonucleotides Bo Wu, Pei Juan Lu, Jignya Ashar, Ehsan Benrashid, Elizabeth Keramaris, Yong Fu Li, Paul A. Morcos, Qi Long Lu. 702 Inhibition of TGF-β Receptor II Expression by Targeting Both Coding and Promoter Regions of the Gene in Hepatic Stellate Cells Jin Wook Kim, Yanhong Zhang, Mark A. Zern, John J. Rossi, Jian Wu. 703 Restoration of Proper Splicing of the β-globin Gene Using Triplex-Forming Oligonucleotides Joanna Y. Chin, Jean Y. Kuan, Faye A. Rogers, Peter M. Glazer. 704 Efficient Organ Selective Non-Viral sirna Delivery System Modified with a Newly Identified Peptide Sequence Erdal Tan, Hidetaka Akita, Diky Mudhakir, Hideyoshi Harashima. 705 Lentiviral RNAi-Vectors Utilizing Pol II-Promoters for Use in the Brain Troels T. Nielsen, Ingrid van Marion, Lis Hasholt, Cecilia Lundberg. 706 Signal-Smart 1 (SS1) Oncolytic Herpes Virus: The First Transcriptionally Targeted Mutant Herpes Virus Designed Against Pro-Oncogenic Signaling Pathways Weihong Pan, Tuba Esfandyari, Farnaz Yamoutpoor, Faris Farassati. FRIDAY, JUNE 1 97

99 Abstract Directory FRIDAY, JUNE 1 Friday, June 1, 2007 Poster Session II continued 5:15 pm 8:15 pm Hall 4AB Oligonucleotide Therapies I continued 707 Inhibiting HIV-1 Gene Expression and Replication with Expressed Long Hairpin RNAs Sheena Saayman, Samantha Barichievy, Kevin V. Morris, Carol Crowther, Patrick Arbuthnot, Marco S. Weinberg. 708 Efficient Delivery and Improved Gene Targeting of PNA- Peptide Conjugates in a Transgenic Mouse Model Faye A. Rogers, Denise Hegan, Leonard Milstone, Peter M. Glazer. 709 Progress on Mutation Independent Silencing of Rhodopsin Using RNA Interference (RNAi) and Trans Complementation by an RNAi-Resistant Rhodopsin Transgene Siobhan M. Cashman, Chikage Kubo, Rajendra Kumar-Singh. 710 Argonaute2 (Ago2) Gene Silencing by Liposomal Transfection with sirna for Ago2 Induces Apoptosis on HT1080 Cells and HUVECs Tatsuhiro Ishida, Tatsuaki Tagami, Jose M. Barichello, Kiyomi Hirose, Naoshi Yamazaki, Tomohiro Asai, Naoto Oku, Hiroshi Kiwada. 711 In Vivo RNA Silencing of the Mcl-1 Gene Confers Increased Susceptibility of Melanoma Cells to Apoptosis Kathleen E. A. Felton, Leah C. Young, Angela M. Keuling, Susan E. Andrew, Victor A. Tron. 712 Gene Editing Mediated by a Novel DNA Analogue Called INA Charlotte B. Sorensen, Anne Krogsdam, Esben N. Flindt, Ulf B. Christensen, Karsten Kristiansen, Thomas G. Jensen, Lars Bolund. 713 Induction of Dystrophin Expression by Exon Skipping in mdx Mice by PNA and PNA-Peptide Conjugates HaiFang Yin, QiLong Lu, Matthew J. A. Wood. 714 Sequence-Specific Inhibition of RNA Polymerase II- and III-Dependent Transcription Using Zorro Locked Nucleic Acid (LNA) Rongbin Ge, Mathias G. Svahn, Oscar E. Simonson, Abdalla J. Mohamed, Karin E. Lundin, Edvard C. I. Smith. 715 Sequence-Specific Triple Helix Formation with Genomic DNA Zhaoyang Ye, Ramareddy V. Guntaka, Ram I. Mahato. 716 Screening of DNAzymes That Inhibit β-catenin Gene Expression in Colon Cancer Cells Bo-Ra Choi, Ki-Sun Kim, Kyoung Jin Jang, Sangtaek Oh, Dong-Eun Kim. 717 Inhibition of Enterovirus 71 in Virus-Infected Mice by RNA Interference Eng Lee Tan, May Chin Theresa Tan, Tak Kwong Vincent Chow, Chit Laa Poh. Saturday, June 2, 2007 Oral Abstract Session 410 AAV Vector Application and Tropism 10:15 am 12:15 pm Room :15 AM Delivery of IL-10 by an AAV Vector to the Lungs of CFTR Knockout Mice Reduces Inflammation upon Chronic Infection with P. aeruginosa Scotty Morrow Buff, Scarlett M. Caldwell, Hong Yu, John E. Baatz, Thomas W. Ferkol, Terence R. Flotte, Isabel L. Virella-Lowell :30 AM Biological Differences in raav Transduction of Human and Old World Non-Human Primate Airway Epithelia Xiaoming Liu, Meihui Luo, Cyndi Trygg, Ziying Yan, Diana C. M. Lei- Butters, Carolina B. Smith, Anne C. Fischer, Keith Munson, William B. Guggino, Bruce A. Bunnell, John F. Engelhardt :45 AM Evaluating AAV-Mediated Transduction of Primate Liver as a Therapeutic Strategy for Lysosomal Storage Disorders Jennifer B. Nietupski, Bradley Hodges, Karen Ashe, Qiuming Chu, Seng H. Cheng, John Marshall, Ronald K. Scheule :00 AM Intravenous Injection Cofactors of Adeno-Associated Virus Produce Varying Tissue-Specific Transduction Levels in Adult Mice Brian R. Schultz, Paul Gregorevic, Eric Finn, Caitlin Doremus, Leonard Meuse, Miki Haraguchi, James M. Allen, Jeffrey S. Chamberlain :15 AM Intravascular Delivery of AAV-9 Leads to Efficient Transduction of Multiple Muscles in Dog Yongping Yue, Janet Bogan, Arka Ghosh, Dan Bogan, Chun Long, Bruce F. Smith, Joe N. Kornegay, Dongsheng Duan. 98

100 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Oral Abstract Session 410 continued AAV Vector Application and Tropism 10:15 am 12:15 pm Room :30 AM Novel AAV Variants Result in Unique Patterns of Transduction in Mouse Brain Cassia N. Cearley, Luk H. Vandenberghe, Erin R. Carnish, James M. Wilson, John H. Wolfe :45 AM Convection-Enhanced Delivery (CED) of AAV1-hrGFP into the Monkey Brain Results in Robust Neuronal and Non-Neuronal Transduction of Cortical and Subcortical Regions Piotr Hadaczek, Hanna Mirek, Keith Munson, John Bringas, Phil Pivirotto, Beverly L. Davidson, Roberto Guerciolini, Krystof Bankiewicz :00 PM Site-Specific Labeling of Targeted AAV with Fluorophores Using Biotin Ligase Allows Comparison of Cellular Binding and Intercellular Trafficking Pathways Used by Modified an Un-Modified Vectors Matthew D. Stachler, Jeffrey S. Bartlett. Oral Abstract Session 411 Adenovirus Vectors: Cancer Therapy 10:15 am 12:15 pm Room 608/ :15 AM Evaluation of Recombinant Mouse Adenovirus Type 1 (MAV- 1) Vectors in Tumor Bearing, Immune Competent Mice Michael J. Robinson, Satya Yendluri, Derek Ko, Ying Ge, Karin Jooss :30 AM Administration of an Osteosarcoma-Targeted Canine Oncolytic Adenovirus in Normal and Tumor-Affected Dogs Bruce F. Smith, David T. Curiel, Vladimir V. Ternovoi, Anton V. Borovjagin, Henry J. Baker, Nancy R. Cox, Annette N. Smith, Mary L. Higginbotham, Gene P. Siegal :45 AM Phase I Study of Intratumoral (IT) Injection with a Telomerase Specific Conditionally Replicative Adenovirus (CRAd), Telomelysin(OBP-301), in Patients with Advanced Solid Tumor Michael Nemunaitis, Neil Senzer, Yu-An Zhang, Alex W. Tong, Jeffrey Lamont, Minal Barve, Hitoshi Kawamura, John J. Nemunaitis :00 AM Long-Term Results of a Phase I/II Study of Ad-OC-TK/VAL Gene Therapy for the Patients with Metastatic or Local Recurrent Prostate Cancer Toshiro Shirakawa, Akinobu Gotoh, Shuji Terao, Nobuyuki Hinata, Kazumasa Goda, Kazushi Tanaka, Atsushi Takenaka, Isao Hara, Chinghai Kao, Thomas A. Gardner, Leland W. K. Chung, Sadao Kamidono, Masato Fujisawa :15 AM Ad16 and CV23 Adenoviruses Are Superior to Adenovirus 5 for Transduction of Human Low-Passage Glioma Cells and Cancer Stem Cells Johan Skog, Karin Edlund, Tommy A. Bergenheim, Göran Wadell :30 AM Carrier Cell-Mediated Delivery of a Replication-Selective Oncolytic Adenovirus for Ovarian Cancer Gene Therapy Katsuyuki Hamada, Yaka Hattori, Ting Zhang, Ayako Nagai, Michihiro Sumida :45 AM Development of Novel Hybrid Adeno-Semliki Forest Viral Vectors for Anti-Cancer Applications Michael L. Roberts, Polyxeni Katsoupi, Teni Boulikas :00 PM Comparative Evaluation of oncolytic Adenoviruses for Selective Eradication of oral Cancer and Precancerous Lesions Hester J. T. van Zeeburg, Ruud H. Brakenhoff, Aafke Huizenga, Zeng B. Zhu, Victor W. van Beusechem. Oral Abstract Session 412 Intracellular Trafficking of Non-Viral Vehicles 10:15 am 12:15 pm Room 4C :15 AM Quantitative Analysis of Intracellular Unpacking of Polymeric DNA Nanoparticles Constructed from Quantum Dot-FRET Hunter H. Chen, Yi-Ping Ho, Xuan Jiang, Hai-Quan Mao, Tza-Huei Wang, Kam W. Leong :30 AM Design of Peptide Vinyl Ester Inhibitors of the Proteasome To Increase Gene Transfer Efficiency Molly E. Martin, Kevin G. Rice :45 AM Glycopolyamines for NF-kappa B Decoy Delivery to Cardiomyocytes Yemin Liu, James Gulick, Jeffrey Robbins, Mike Tranter, Suiwen He, W. Keith Jones, Theresa M. Reineke :00 AM Novel Layer-by-Layer Polyplexes Using Poly(Ethylenimine) and Poly(Acrylic Acid) for Gene Delivery Chung-Huei K. Wang, Justin M. Saul, Chee P. Ng, Suzie H. Pun. SATURDAY, JUNE 2 99

101 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Oral Abstract Session 412 continued Intracellular Trafficking of Non-Viral Vehicles 10:15 am 12:15 pm Room 4C :15 AM Targeted Delivery of Therapeutic Nucleic Acids to Breast and Pancreatic Cancer Tumors Using Multifunctional Nanoparticles with Activity Modulated by Redox Potential Gradients Devika Soundara Manickam, Aiko Hirata, David Putt, Fazlul Sarkar, Lawrence Lash, Fusao Hirata, David Oupicky :30 AM A Novel Peptide Discovered by Intracellular Bacteriophage Display Mediates Nuclear Targeting Andrija Finka, Daniel C. Dorset, Jeffrey A. Hubbell, Todd D. Giorgio :45 AM Chromatin Reconstitued with Engineered Histones as an Efficient Gene Delivery Agent Kylie M. Wagstaff, Jun Y. Fan, Michelle A. De Jesus, David J. Tremethick, David A. Jans. 741 Moved to Poster Session II after Abstract 524 Oral Abstract Session 413 Neurologic II 10:15 am 12:15 pm Room :15 AM Intracerebral AAV5-Mediated Gene Transfer in Metachromatic Leukodystrophy: Preclinical Evaluation of Efficiency and Tolerance in Large Animal Caroline Sevin, Marie-Anne Colle, Francoise Piguet, Lise Bertrand, Sylvie Raoul, Christophe Darmon, Laetitia Jeanneau, Francoise Fouquet, Jerome Amiaud, Yan Cherel, Philippe Moullier, Patrick Aubourg, Nathalie Cartier :30 AM Dose Response of Subretinal AAV2 and AAV8 GFP Gene Transfer to the Macaque Macula: Favorable Safety Profile but Risk of Focal Immunotoxicity at the Highest Dose Luk H. Vandenberghe, Peter Bell, Roberto Calcedo, Lili Wang, Rebecca Grant, Tracie E. C. Irvin, Tim Hopkins, Albert M. Maguire, James M. Wilson, Jean Bennett :45 AM In Vivo Engineering of Light Sensitive Neurons in the Diseased Retina Kenneth P. Greenberg, Katharine Borges, Dirk Trauner, Richard H. Kramer, John G Flannery :00 AM Reversal of Visual Deficits in Animal Models of Leber Congenital Amaurosis within Weeks after Treatment with an Optimized Adeno-Associated Virus (AAV) Jean Bennett, Jeannette Bennicelli, Fraser Wright, Valder R. Arruda, Olga Zelenaia, Berndt Hauck, Tonia S. Rex, Daniel Chung, Zhangyong Wei, Kristina Narfstrom, Edward N. Pugh, Jr, Jonathan Jacobs, Lou Dell Osso, Caroline Zeiss, Andras M. Komaromy, Gregory M. Acland, Katherine A. High, Albert M. Maguire :15 AM AAV-Mediated Gene Therapy of Albinism Ciro Bonetti, Annagiusi Gargiulo, Pasqualina Colella, Sandro Montefusco, Claudio Mussolino, Umberto Di Vicino, Andrea Ballabio, Alberto Auricchio, Enrico Maria Surace :30 AM Systemic Insulin-Like Growth Factor-1 (IGF-1) Therapy Reverses Hyposensitivity in a Mouse Model of Diabetic Peripheral Neuropathy (DPN) Qiuming Chu, Rod Moreland, Nelson S. Yew, Seng H. Cheng, Ronald K. Scheule :45 AM Impact of Age of Treatment on AAVrh.10hCLN2-Mediated Amelioration of Symptoms in CLN2 Knockout Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis Dolan Sondhi, Neil R. Hackett, Jocelyn Ronda, Michael Baad, Ronald G. Crystal :00 PM SMA Based Therapeutic Trans-Splicing RNAs Repair SMN1 Deficiency Tristan H. Coady, Monir Shababi, Chris L. Lorson. Oral Abstract Session 414 Vaccination Via Gene Delivery 10:15 am 12:15 pm Room 4C :15 AM A Vectored Measles Vaccine Induces Hepatitis B Surface Antigen Antibodies While Protecting Macaques Against Measles Challenge Jorge Reyes del Valle, Patricia Devaux, Nicholas J. Wegner, Gregory Hodge, Michael B. McChesney, Roberto Cattaneo :30 AM 4-1BB Antibody Agonist Significantly Enhances SIV-Specific CD8+ T-Cell Immunity and Proliferation Following Naked DNA Vaccination in Cynomolgus Macaques David A. Hokey, Lauren A. Hirao, Sandra A. Calarota, Jian Yan, Jean D. Boyer, Anlan Dai, Hanna Yoon, Maria N. Jure-Kunkel, Praveen Balimane, David B. Weiner. 100

102 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Oral Abstract Session 414 continued Vaccination Via Gene Delivery 10:15 am 12:15 pm Room 4C :45 AM Comparison of the Efficacy of a Six Genetic Anti-Plaque Vaccine Candidates Against a Lethal Respiratory Tract Challenge with Yersinia pestis Julie L. Boyer, Steven Park, Maria Chiuchiolo, Svetlana Senina, William R. Jacobs, Ralph M. Steinman, Yoonkyung Do, Gyu Park, John K. Rose, Jorge E. Galan, Peter Palese, David Perlin, Ronald G Crystal :00 AM Single Administration of an Adenovirus Vectored Molecular Vaccine Provides Fast-Acting Protection Against Foot-and- Mouth Disease Virus in Cattle Douglas E. Brough, David Brake, Damodar Ettyreddy, John Neilan, Lazlo Zsak, Jose Barrera, William T. Golde, Femi Olutunmbi, Aniko Zsak, Rick King, Luis L. Rodriguez, Mauro P. Moraes, Marla Koster, Marvin J. Grubman :15 AM Enhanced Immunogenicity to HIV Plasmid Vaccines in Non- Human Primates Using an IL-12 Expressing Plasmid and Constant-Current Electroporation Lauren Hirao, Amir S. Khan, Ruxandra Draghia-Akli, David B. Weiner :30 AM Induction of Both Rapid and Persistent Passive Immunity Against Anthrax Toxin by Co-Administration of Adenovirus and Adeno-Associated Virus Type rh.10 Vectors Expressing Anti-Protective Antigen Antibody Bishnu P. De, Julie L. Boyer, Neil R. Hackett, JianPing Qiu, James M. Wilson, Ronald G. Crystal :45 AM Vaccination with Linear Expression Cassettes Protects Mice Against Lethal Influenza A Challenge Adrian Vilalta, Gretchen Jimenez, Denis Rusalov, Peggy Lalor, Kristin Carner, Jennifer A. Chaplin, Michael Komai, Marston Manthorpe, Alain Rolland, David C. Kaslow :00 PM Generation of Protective Immunity Against 1918 (H1N1) and Divergent Isolates of Avian Influenza (H5N1) Viruses through Different Vaccination Strategies Darwyn Kobasa, Ami Patel, Michael Gray, Gary P. Kobinger. Oral Abstract Session 415 Cancer Gene Therapy 10:15 am 12:15 pm Room 606/ :15 AM Adenoviral-Mediated Delivery of Pseudomonas Exotoxin (PE) Fused to IL-13 Induces Regression of Intracranial Human Glioblastoma Xenografts in Mice M. Candolfi, W. Xiong, M. Puntel, A. K. M. G. Muhammad, Chunyan Liu, Kurt M. Kroeger, Sonali Monkar, Ron Rodriguez, Pedro R. Lowenstein, Maria G. Castro :30 AM Lymphoma Chemovirotherapy: CD20-Targeted and Convertase-Armed Measles Virus Enhances Fludarabine Efficacy Locally Guy Ungerechts, Christoph Springfeld, Marie E. Frenzke, William B. Parker, Eric J. Sorscher, Roberto Cattaneo :45 AM Effects of Stable Endostatin and Angiostatin Gene Therapy on Angiogenesis-Related Gene Expression in Multistage Prostate Cancer in TRAMP Model Tatyana Isayeva, Diptiman Chanda, Dongquan Chen, Selvarangan Ponnazhagan :00 AM Retroviral Vector-Producing Mesenchymal Stem Cells for Tumor Tracking and Therapeutic Gene Amplification as Systemic Suicide Cancer Gene Therapy Ryosuke Uchibori, Takashi Okada, Takayuki Ito, Takashi Matsushita, Masashi Urabe, Hiroaki Mizukami, Akihiro Kume, Keiya Ozawa :15 AM Transcriptional Targeting Achieves Effective Control of Systemic Dissemination by Replication-Competent Retrovirus Takahiro Kimura, Kazunori Haga, Christopher R. Logg, Kei Hiraoka, Greg W. Lawson, Robert J. Matusik, Bernard H. Bochner, Shin Egawa, Renata Stripecke, Noriyuki Kasahara :30 AM Mutual Sensitization with Telomerase-Specific Oncolytic Virotherapy and Ionizing Radiation in Cytotoxic Activity Against Human Cancer Shinji Kuroda, Toshiya Fujiwara, Yuichi Watanabe, Yuuri Hashimoto, Toru Kojima, Ryo Sakai, Masaaki Ouchi, Hideki Onimatsu, Yasuo Urata, Shunsuke Kagawa, Noriaki Tanaka, Toshiyoshi Fujiwara :45 AM Enhancing the Therapeutic Index of Oncolytic CVA21 Elizabeth Hadac, Elizabeth Kelly, Stephen J. Russell. SATURDAY, JUNE 2 101

103 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Oral Abstract Session 415 continued Cancer Gene Therapy10:15 am 12:15 pm Room 606/ :00 PM Nature of Seneca Valley Virus (SVV-001/NTX-010) RNA in Blood after Systemic Delivery to Patients with Solid Tumors Expressing Neuroendocrine Features Pranay D. Khare, P. Seshidhar Reddy, Kevin D. Burroughs, Grace Johnston, Neeraja Idamakanti, Brian H. Jones, Shawn Li, Ann-Jeanette Vasko, Kristine L. Skele, Chris Sherk, Jennifer Harris, Laura M. Hales, John R. Neefe, Paul L. Hallenbeck. Oral Abstract Session 416 Hematopoietic Stem Cell Gene Therapy 10:15 am 12:15 pm Room 4C1/4C :15 AM Exploring the Influence of Retroviral Integration Site Selection on Gene Expression in ADA-SCID Patients Treated with Stem Cell Gene Therapy Barbara Cassani, Eugenio Montini, Fabrizia Urbinati, Grazia Andolfi, Massimiliano Mirolo, Luca Biasco, Alessandra Recchia, Samantha Scaramuzza, Matteo Cazzola, Daniela Sartori, Alessandro Ambrosi, Clelia Di Serio, Maria Grazia Roncarolo, Luigi Naldini, Fulvio Mavilio, Claudio Bordignon, Alessandro Aiuti :30 AM Therapeutic Expression of a Platelet-Specific Integrin Restores Hemostasis in Dogs with Glanzmann Thrombasthenia David A. Wilcox, Juan Fang, Eric S. Jensen, Lily M. Du, Mary K. Boudreaux :45 AM Transplantation of Genetically Modified Cells for Treatment of Infection in Patients with X-Linked Chronic Granulomatous Disease (X-CGD) Elizabeth M. Kang, Gilda Linton, Narda Theobald, Effie Nomicos, Dianne Hilligoss, Harry L. Malech :00 AM Correction of Canine XSCID by Gene Therapy Using Preclinical SIV Lentiviral Vectors Expressing Human or Canine Gamma Chain Morvarid Moayeri, Douglas R. Kennedy, Nora Naumann, Yasuhiro Ikeda, Felsburg J. Peter, Harry L. Malech, Suk See De Ravin :15 AM Self-Inactivating Retroviral Vectors Expressing hil2rg from an Internal SFFV Enhancer-Promoter Induce Leukemia by Insertional Mutagenesis Ute Modlich, Axel Schambach, Daniel Wicke, Sabine Knoess, Christopher Baum :30 AM Successful Gene Therapy of Canine Leukocyte Adhesion Deficiency Using Foamy Viral Vectors Thomas R. Bauer, Jr, James M. Allen, Laura M. Tuschong, Erik M. Olson, Tanya H. Burkholder, Mehreen Hai, Yu-chen Gu, Dennis D. Hickstein, David W. Russell :45 AM Efficient Transduction of Macaque Hematopoietic Repopulating Cells with HIV-Derived Lentiviral Vectors Grant D. Trobridge, Brian C. Beard, Philip Olsen, Punam Malik, Hans- Peter Kiem :00 PM Clinically Relevant Levels of Hematopoietic Stem Cell Gene Transfer in Rhesus Macaques Nearly Four Years Following Transplantation with Autologous Hematopoietic Cells Transduced with an Simian Immunodeficiency Virus-Based Lentiviral Vector Yoon Sang Kim, Yoon-Jin Kim, Peiman Hematti, Bum Kee Hong, Donahue Robert, Arthur W. Nienhuis, Cynthia E. Dunbar, Derek A. Persons. Oral Abstract Session 417 Gene Modification 10:15 am 12:15 pm Room :15 AM Understanding and Exploiting Endogenous microrna Regulation for Controlling Gene Expression Brian D. Brown, Alessio Cantore, Bernhard Gentner, Anna Zingale, Luigi Naldini :30 AM The Woodchuck Hepatitis Virus Post-Transcriptional Regulatory Element Reduces Readthrough Transcription from Retroviral Vectors To Improve Vector Titers and Expression Tomoyasu Higashimoto, Fabrizia Urbinati, Anil Perumbeti, Gang Jiang, Alexander Zarzuela, Lung-Ji Chang, Donald B. Kohn, Punam Malik :45 AM An Engineered Zinc Finger Protein Transcriptional Activator of Pigment Epithelium-Derived Factor Drives Long-Term Inhibition of Choroidal Neovascularization H. Steve Zhang, Katsutoshi Yokoi, Shinji Ueno, Naw H. Khu, Qi Yu, Richard Surosky, Liza M. Africa, Philip D. Gregory, Peter A. Campochiaro. 102

104 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Oral Abstract Session 417 Gene Modification continued 10:15 am 12:15 pm Room :00 AM Targeted Gene Addition into a Putative Safe-Harbor Locus in the Human Genome Using Engineered Zinc Finger Nucleases Russell C. DeKelver, Erica A. Moehle, Jeffrey C. Miller, Jeremy M. Rock, David E. Paschon, Igor Rupniewski, Phillip Y. Tam, Edward J. Rebar, Michael C. Holmes, Philip D. Gregory, Fyodor D. Urnov :15 AM Identification of Additional Barrier and Protein Binding Activities in Extended Core Sequences of the chs4 Chromatin Insulator Mari Aker, Amy C. Groth, Gary Felsenfeld, Hans-Peter Kiem, George Stamatoyannopoulos, David W. Emery :30 AM Gaussia Luciferase for Monitoring of Tumor Growth and Therapy in Culture and In Vivo Thomas Wurdinger, Lisa Pike, Ralph Weissleder, Xandra O. Breakefield, Bakhos Tannous :45 AM Controlling T-Cell Proliferation and Survival with Synthetic, Small Molecule-Controlled RNA-Based Regulatory Systems Yvonne Y. Chen, Christine E. Brown, Michael C. Jensen, Christina D. Smolke :00 PM The DNA-Binding Specificity of Designer Zinc Finger Nucleases Is a Major Determinant of Activity and Genotoxicity in Human Cells Tatjana I. Cornu, Stacey Thibodeau-Beganny, Eva Guhl, Stephen Alwin, Magdalena Eichtinger, J. Keith Joung, Toni Cathomen. Poster Session III 5:15 pm 7:00 pm Hall 4AB RNA Virus Vectors: Novel Vectors and Applications 782 Characterizing the Molecular Mechanism for Robust, Sustained Hepatic Expression from Episomal Lentiviral Vectors Matthew A. Bayer, Brian Zeithaml, Boris Kantor, Thomas McCown, Hong Ma, Tal Kafri. 783 Systemic Comparison of Promoters Driving Transgene Expression in Airway Epithelia Using a Non-Primate Lentiviral Vector Erin R. Burnight, Patrick L. Sinn, Paul B. McCray. 784 Differential Regulation of Interferon Alpha-Responsive Antiviral Pathways Determines Tumor Selectivity of Newcastle Disease Virus Subbiah Elankumaran, Daniel D. Rockemann, Siba K. Samal. 785 Lentiviral Protein Transduction and Intracellular PIC Visualization by Vector Incorporated HIV-1 Integrase Fusion Proteins Diana A. M. Schenkwein, Vesa P. Turkki, Hanna P. Lesch, Karkkainen A. Hanna-Riikka, Mahonen J. Anssi, Airenne J. Kari, Yla-Herttuala Seppo. 786 Optimizing Systemic Delivery of Oncolytic Measles Virus by Using Virus Infected Cell Carriers Kah-Whye Peng, Hooi T Ong, Allan B. Dietz, Stephen J. Russell. 787 Non-Transmissible SeV Encoding Murine GM-CSF, Another Potent Vector System To Produce Autologous Tumor Vaccines Hiroyuki Inoue, Takafumi Nnakamura, Terumasa Hisano, Meng Xin, Saori Asahi, Ryo Kurita, Koichi Takayama, Yoshikazu Yonemitsu, Makoto Inoue, Mamoru Hasegawa, Yoichi Nakanishi, Kenzaburo Tani. 788 Development of Analytical Tools in Downstream Processing of Influenza Virus Johanna Norberg, Mia Bangtsson, Anita Larsson, Camilla Estmer Nilsson. 789 Inhibition of HIV-1 Replication by a Combination of RNaseP and 3 trnase-associated External Guide Sequences (EGSs) in Mammalian Cells Yuichiro Habu, Naoko Miyano-Kurosaki, Hiroshi Takaku. 790 Transduction of Primary Human Monocytes/Macrophages Using HIV-1-Derived Vector Particles Is Enhanced by the Viral Protein Vpx PBj Provided by SIV sm PBj-Derived Vector Like-Particles Silke Schüle, Nina Wolfrum, Julia Kaiser, Björn-Philipp Kloke, Klaus Cichutek, Matthias Schweizer. 791 Macrophage Differentiation from Human CD34 Cells in order To Test Anti-HIV Vectors Jason A. Taylor, Lucia Vojtech, Brent L. Wood, David W. Russell, Robert E. Richard. 792 Biodistribution and Tumor Infectivity of Semliki Forest Virus Vectors in Mice: Effects of Readministration Juan R. Rodriguez-Madoz, Jesus Prieto, Cristian Smerdou. SATURDAY, JUNE 2 103

105 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB RNA Virus Vectors: Novel Vectors and Applications continued 793 Integration-Defective Lentiviral Vectors as a Platform for Sleeping Beauty Transposase-Mediated Gene Insertion Nicklas H. Staunstrup, Maria Jakobsen, Brian Moldt, Jacob G. Mikkelsen. 794 Development of Novel Defective Sendai Virus Vectors Capable of Expressing Therapeutic Genes Persistently without Chromosomal Integration Ken Nishimura, Hiroaki Segawa, Mahito Nakanishi. 795 Efficient In Vivo Transduction Using RNA Replicon Vector Derived from Sendai Virus Self-Replicating Ribonucleoprotein Complexes Takashi Hironaka, Makoto Inoue, Eisaku Suzuki, Hitoshi Iwasaki, Kentaro Washizawa, Satoshi Fujikawa, Akihiro Iida, Mamoru Hasegawa. 796 In Vitro Evolution of a Replicating, Chimeric Murine Leukemia Virus-Gibbon Ape Leukemia Virus Vector Christopher R. Logg, Brian T. Baranick, Nathan A. Lemp, Noriyuki Kasahara. 797 Regulated HIV-1 and HIV-2 Lentiviral Vectors Suresh K. Arya, Adriana Zingone, Satomi Adaka, Geetanjali Sachdeva. 798 Expression of Transduced Proteinase Inhibitor 9 Is Upregulated in Human Islets Exposed to Pro-Apoptotic Stimuli; Implications for Protection of Transplanted Islets Sirlene Cechin, M. Sofia Ochoa, Ingrid Perez-Alvarez, Michael A. Curran, Elizabeth S. Fenjves. 799 Optimizing a Protocol for Transduction of Nonhuman Primate ES Cells with Lentiviral Vectors Reeteka Sud, Szczepan W. Baran, Philip M. Olsen, Angelique M. Nelson, Carol B. Ware, Darlene A. Pedersen, Shoukhrat M. Mitalipov, Hans-Peter Kiem. 800 MGMTP140K Allows for Efficient and Sustained In Vivo Selection in Baboons and Macaques Brian C. Beard, Grant D. Trobridge, Philip M. Olsen, James Fletcher, Kate Beebe, Julia Piasecki, Hans-Peter Kiem. 801 Hepatic Expression of MGMT P140K Enables Selection in Primary Hepatocytes: Implication in Gene Therapy for MPS Type I in Mice Daren Wang, Nicole Worsham1, Dao Pan. 802 Lenti-Vector Optimization for IDUA Expression in the Murine Model of Hurler Syndrome (Mucopolysaccharidosis Type I) Paul R. Score, Arthur T. Johnson, Chester B. Whitley. 803 Optimization of the Stem and Loop of shrna Lentiviral Constructs for Stable RNA Interference into Human Hematopoietic Cells Laurence Jeanson-Leh, Johanna Blondeau, Anne Galy. RNA Vector Production and Targeting 804 Targeting of Lentiviral Vectors by Magnetic Nanoparticles Andreas Hofmann, Lars Burdorf, Johannes Postrach, Daniela Wenzel, Jutta Weisser-Thomas, Diana Messow, Bernhard Gleich, Thomas Weyh, Bruno Reichart, Bernd K. Fleischmann, Michael Schmoeckel, Alexander Pfeifer. 805 Retroviral Particle Mediated mrna Transfer: New Insights into Underlying Mechanisms Melanie Galla, Axel Schambach, Elke Grassman, Zoltan Ivics, Greg Towers, Christopher Baum. 806 Psuedotyping Lentiviral Vector Particles for the Envelope Protein from Feline Leukemia Virus Type C (FLVC) Matthew Wielgosz, Phillip Hargrove, Steven Kepes, John Gray, Derek Persons, Arthur Nienhuis. 807 Engineering the Tropism of VSVG-Pseudotyped Retroviral and Lentiviral Vectors Via the Insertion of RGD Targeting Peptides Kwang-il Lim, Julie Yu, David Schaffer. 808 The Two Phases of Measles Virus Cell Entry through SLAM: Hemagglutinin Residues Necessary for Primary Binding or Membrane Fusion Chanakha K. Navaratnarajah, Sompong Vongpunsawad, Numan Oezgun, Rachel Trehin, Thilo Stehle, Werner Braun, Roberto Cattaneo. 809 Two Viruses: Similar Fibers, Similar Receptors, Different Destinies Katherine J. D. A. Excoffon, Nicholas D. Gansemer, J. Denise Wetzel, Kristen M. Guglielmi, Jacquelyn A. Campbell, Joseph Zabner, Terence S. Dermody. 104

106 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB RNA Vector Production and Targeting continued 810 Increasing Incorporation of HN Enhances Infectivity of HPIV3-Pseudotyped Lentiviruses Cindy Jung, Joseph M. Le Doux. 811 Titers of HIV-Based Vectors Encoding shrnas Are Reduced by a Dicer-Dependent Mechanism Anantha Lakshmi Poluri, Richard E. Sutton. 812 Composition of Purified Lentiviral Vector Products Intended for Gene Therapy: Implications for Quality Control of Vector Production Kenneth Keating, Carl Dolman, Robin Thorpe, Yuan Zhao. 813 Cell Entry Targeting of Lentiviral Vectors through Pseudotyping with the Measles Virus H and F Proteins Sabrina Funke, Andrea Maisner, Roberto Cattaneo, Klaus Cichutek, Christian J. Buchholz. 814 The Thermo-Sensitivity of the Reverse Transcription Process Is a Mechanism of MLV-Based Vectors Inactivation Marlene Carmo, Amos Panet, Manuel J. T. Carrondo, Paula M. Alves, Pedro E. Cruz. 816 Improved Production Protocols for HIV-1-Based Lentiviral Vectors Robert H. Kutner, Jakob Reiser. 817 A Quantitative Kinetic Analysis of Centrifugation-Based Delivery of Retroviral Vectors into Mammalian Cells Venkata S. Tayi, Pascal R. Beauchesne, Bruce D. Bowen, James M. Piret. 818 Development of Scaleable Retroviral Transduction Strategies Utilizing Whole Cell Lysate and Acoustic Technology Pascal R. Beauchesne, Venkata S. Tayi, Bruce D. Bowen, James M. Piret. AAV Application I 819 Evaluation of Hybrid Adeno-Associated Virus (AAV) Vectors for Transduction of Cells with Primary Cilia: Applications for Retinal, Cochlear, and Renal Disease Daniel C. Chung, Jeff Bedrosian, Zhangyong Wei, Luk H. Vanderberghe, Gao Ping, James M. Wilson, Michael A. Gratton, Joshua H. Lipschutz, Jean Bennett. 820 Evaluation of an Adenoassociated Viral Vector Construct Containing the First 26 Amino Acids of CFTR but Missing the First 4 Loops of TMD1 Liudmila Cebotaru, Terence R. Flotte, William B. Guggino. 821 The AAV9 Capsid Preferentially Transduces Cardiac Tissue and Demonstrates Unique Behavior In Vivo Christina A. Pacak, Cathryn S. Mah, Bijoy Thattaliyath, Melissa A. Lewis, Sean Germain, Glenn A. Walter, Barry J. Byrne. 822 Comparative Analysis of Multiple AAV Vector Serotypes in the Mouse Brain Jean-Marc Taymans, Luk H. Vandenberghe, Chris Van Den Haute, Irina Thiry, Christophe M. Deroose, Luc Mortelmans, James M. Wilson, Zeger Debyser, Veerle Baekelandt. 823 In Vitro Identification of Optimal Exon-Skip Targets for Treatment of Duchenne Muscular Dystrophy Using a Human Muscle Cell Line Nathaniel A. Walton, Clifford J. Beall, Jerry R. Mendell, K. Reed Clark. 824 Significantly Extended Survival and Improved Behaviore of MPS IIIB Mice by an Intracisternal Delivery of raav2 Vector Haiyan Fu, Julianne DiRosario, Lu Kang, Jerillyn S. Jennings, Joseph Muenzer. 825 Gene Delivery to Intestinal Epithelial Cells In Vitro and In Vivo with Recombinant Adeno-Associated Virus Types 1, 2 and 5 Steven F. Polyak, Cathryn Mah, John D. Herlihy, Stacy L. Porvasnik, Martha Campbell-Thompson, John F. Valentine. 826 Novel AAV Serotypes Result in Efficient Photoreceptor Transduction Mariacarmela Allocca, Maria Garcia Hoyos, Luk H. Vandenberghe, Carolina Iodice, Claudio Mussolino, Marco Petrillo, James M. Wilson, Enrico M. Surace, Alberto Auricchio. 827 Secretion of IL-10 from Recombinant Skin Generated by AAV2/IL-10 Transduction Sarmistha Bandyopadhyay, Yong Liu, Jawahar L. Mehta, Paul L. Hermonat. 828 Gene Transfer of Superoxide-Dismutase Genes and Catalase Confers Radioprotection on Normal Human Primary Lung Fibroblasts and Lymphoblastoid Cells Marlon R. Veldwijk, Carsten Herskind, Patrick Maier, Stefan Fruehauf, W. Jens Zeller, Frederik Wenz. SATURDAY, JUNE 2 105

107 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB AAV Application I continued 829 Adeno-Associated Virus-Mediated Gene Transfer: The Combined Use of Self-Complementary AAV Vectors and the Parvovirus B19 Promoter Leads to Expression of Therapeutic Levels of the Human Beta-Globin Gene Njeri Maina, Weihong Zhao, Xiaomiao Li, Zongchao Han, Li Zhong, George Aslanidi, Sergei Zolotukhin, William Slayton, Arun Srivastava. 830 Gene Transfer to Murine Substantia Nigra Using Adeno- Associated Viral Vectors Pseudo-Typed with Capsids from Serotypes 7 and 9 Douglas B. Howard, Chi-Chung Kuo, Yun Wang, Brandon K. Harvey. 831 AAV Serotype/Genome Comparison for Gene Therapy Targeting the Liver Astrid Paneda, Vanrell Lucia, Itsaso Mauleon, Julien S. Crettaz, Pedro Berraondo, Eric J. Timmermans, Jaap Twisk, Sander van Deventer, Jesus Prieto, Antonio Fontanellas, Rodriguez-Pena Maria Sol, Gonzalez- Aseguinolaza Gloria. 832 The IL-2 and IL-12 Genes Favor Paracrine over Autocrine- Targeted AAV2 Gene Delivery in the Generation of Cytotoxic T Lymphocytes, in Fact AAV/IL-2 Autocrine Delivery Is Strongly Inhibitory Yong Liu, Min Shi, Junaid Khan, Paul L. Hermonat. 833 Efficient Whole Body Muscle Transduction with Self- Complementary AAV 9 Vectors Following Systemic Delivery in Adult Mice Christel Riviere, Valerie Allo, Karine Poulard, Patrice Denefle, Anne M. Douar. 834 AAV8 Vectors Transduce Oligodendrocytes Efficiently Shin-ichi Muramatsu, Hiroko Nishida, Yuko Nara, Naomi Takino, Sayaka Asari, Mika Kodera, Wei-zhong Xiao, Yasuo Sasaki, Satoru Kikuchi, Takashi Matushita, Takashi Okada, Minako Hoshi, Imaharu Nakano, Keiya Ozawa. 835 Self-Complementary Adeno-Associated Virus Serotypes Can Induce Efficient Long-Term Therapeutic Effects in Human Cancer Cells Han Saem Lee, Oh Kyo Shin, Sung Jin Kim, Won Il Lee, Ji Yun Kim, Sunjoo Jeong, Keerang Park, Han Choe, Heuiran Lee. 836 Seropositivity Against AAV Serotypes 1, 8 and 9 in Cynomolgus Monkey Colonies Hiroaki Mizukami, Akira Ishiwata, Fumiko Ono, Jun-ichiro Takano, Koji Fujimoto, Jun Mimuro, Masashi Urabe, Akihiro Kume, Keiji Terao, Yoichi Sakata, Keiya Ozawa. 837 Long-Term Effect and Biosafety of Recombinant AAV-Rat Cygb Expression Ruian Xu, Xinyan Li, Phillip Harriosn, Weidong Xiao, Nagy Habib, Farzin Farzaneh. 838 AAV2 Capsid Specific CTLs Do Not Eliminate AAV Vector Transduced Cells In Vivo Chengwen Li, Matt Hirsch, Aravind Asokan, Brian Zeithaml, Hong Ma, Tal Kafri, Richard Jude Samulski. AAV Application II 839 Distinct Immunoglobulin G Subclass Responses Following Natural Exposure or Vector Infusion of Adeno-Associated Virus in Humans Samuel L. Murphy, Hojun Li, Shyrie Edmonson, Katherine A. High. 840 Liver Directed AAV-Mediated Homologous Recombination Is Independent of Serotype Karsten Wursthorn, Nicole Paulk, Terry Storm, Milton Finegold, Mark Kay, Markus Grompe. 841 Gender as a Factor for Gene Transfer to Rodent Salivary Glands Antonis Voutetakis, Changyu Zheng, Jianghua Wang, Corinne M. Goldsmith, Martin L. Wenk, Molly Vallant, Richard D. Irwin, Bruce J. Baum. 842 Gene Correction by Different Repair Substrates Matthew L. Hirsch, Richard J. Samulski. 843 Interaction of Host and Vector-Dependent Factors in Determining the Risk of Germline Transmission of AAV Vectors Patricia Favaro, Harre D. Downey, Federico Mingozzi, Frederico Xavier, Fraser Wright, Katherine High, Valder R. Arruda. 844 Differential Immune Responses to Transgene Products from raav1 and raav8 Vectors Yuanqing Lu, Sihong Song. 106

108 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB AAV Application II continued 845 Characterization of a Bipartite raav Vector for Site Specific Integration Chun Zhang, Nenita Cortez, Kenneth I. Berns. 846 Strategies for Improving Adeno-Associated Virus Vector- Mediated Gene Transfer in Primary Murine Hematopoietic Stem Cells In Vivo Njeri Maina, Zongchao Han, Xiaomiao Li, Weihong Zhao, Li Zhong, Zhongbo Hu, Wenqin Ma, William Slayton, Arun Srivastava. 847 Differential Uptake and Nuclear Uncoating of Adeno- Associated Virus (AAV) Vectors as Determinats of Transduction in Cells of Cardiac Origin Isaac Sipo, Sandra Pinkert, Lennart Suckau, Xiaomin Wang, Stefan Weger, Henry Fechner, Wolfgang Poller. 848 Hybrid Adeno-Associated Virus Bearing Nonhomologous Inverted Terminal Repeats Enhances Dual-Vector Reconstruction of Minigenes In Vivo Ziying Yan, Diana C. M. Lei-Butters, Yulong Zhang, Roman Zak, John F. Engelhardt. 849 Increased Complexity of Wild-Type AAV-Chromosomal Junctions as Measured by Analysis of Whole Unselected Cellular Genomes: Implications for New Vector Design Horace R. Drew, Linda J. Lockett, Gerald W. Both. 850 Effect of Animal Age and the Route of Administration on Systemic AAV-9 Transduction Brian P. Bostick, Arkasubhra Ghosh, Yongping Yue, Chun Long, Donsheng Duan. 851 The Effect of Genome Size and Design of scaav Vectors on Efficiency of shrna Expression and Gene Knockdown Lennart Suckau, Roland Vetter, Jens Kurreck, Sandra Pinkert, Henry Fechner, Wolfgang Poller. 852 AAV Rep78 Interacts with the E1 Helper Protein of HPV and Enhances Its ATPase Activity Sarmistha Bandyopadhyay, Kevin D. Rainey, Yong Liu, Paul L. Hermonat. 853 Characteristics of Transduction by Self-Complementary Adeno-Associated Virus Serotypes in Human Monocyte- Derived Dendritic Cells OhKyu Shin, SungJin Kim, WonIl Lee, JiYun Kim, Heuiran Lee. 854 Evaluation of Hybrid Adeno Associated Virus (AAV)-Mediated Transduction of the Fetal Retina: Impact of Single Amino Acid Changes of the Capsid Tonia S. Rex, Vivian Lee, Luke H. Vandenberghe, Dan Chung, Zhangyong Wei, Jim M. Wilson, Jean Bennett. 855 Optimization of Delivery and Expression of the Pyruvate Dehydrogenase E1 Alpha Subunit Gene Using Self- Complementary Adeno-Associated Virus Serotype Vectors Zongchao Han, Kristen Berendzen, Li Zhong, Nitin Chouthai, Weihong Zhao, Njeri Maina, Arun Srivastava, Peter Stacpoole. Adenovirus Vectors: Targeting and Host Response 856 Measuring Clearance of Adenovirus Vectors from the Circulation by Kupffer Cells: Inhibition by Polyanions Zhili Xu, Jeffrey S. Smith, Andrew P. Byrnes. 857 The Effect of Coagulation Factors on Ad19p Fiber Mediated Transduction Laura Denby, Lorraine M. Work, John H. McVey, Stuart A. Nicklin, Andrew H. Baker. 858 Biodistribution, Persistence, Innate Immune Response and Toxicity with Nonhuman Adenoviral Vectors in the Mouse Model Anurag Sharma, Dinesh S. Bangari, Aseem Pandey, Harm HogenEsch, Suresh K. Mittal. 859 Regulation of Adenovirus Infection by a Novel Protein, CAR- Like Soluble Protein (CLSP) Kenji Kawabata, Fuminori Sakurai, Katsuhisa Tashiro, Naoki Osada, Jun Kusuda, Takao Hayakawa, Hiroyuki Mizuguchi. 860 Modification of the Ad35 Fiber To Ablate CD46 Binding David A. Einfeld, Cheng Cheng, Douglas E. Brough, Christopher D. Reiter, Megan Fultz, Anne Bet, C. Richter King, Gary J. Nabel, Gall G. D. Jason. 861 Adenovirus Serotype 35 Vector-Mediated Gene Transfer into Cynomolgus Monkeys Hiroyuki Mizuguchi, Fuminori Sakurai, Kimiyo Akitomo, Shin-ichiro Nakamura, Hiroaki Shibata, Keiji Terao, Kenji Kawabata. SATURDAY, JUNE 2 107

109 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Adenovirus Vectors: Targeting and Host Response continued 862 A Genetically Modified Adenoviral Vector for Lung-Selective Gene Expression Joel N. Glasgow, Masaharu Nakayama, Meredith A. Preuss, Raymond L. Benza, David T. Curiel. 863 The KKTK Motif in the Human Adenovirus Serotype 5 Fiber Shaft Domain Is Not Essential for Efficient Virus Infection of Liver Cells In Vivo Nelson C. Di Paolo, Oleksandr Kalyuzhniy, Dmitry M. Shayakhmetov. 864 Differential Biodistribution and Physical Properties of Chimeric Ad5/40S Are Associated to Ad40 Short Fiber Ester Rodriguez, Carol Romero, Mireia Ferrer, Joan F. Burgueno, Adolfo del Rio, Assumpcio Bosch, Estel Gil, Hirofumi Hamada, Ester Fernandez, Miguel Chillon. 865 Targeting Adenovirus Vector to Neural Cell Adhesion Molecule (NCAM) for Gene Delivery to Developing Muscle Bhanu Munil Koppanati, Christoph Volpers, Joel Kaar, Florian Kreppel, Alan J. Russell, Stefan Kochanek, Carl Lagenaur, Paula R. Clemens. 866 Development of Peptide Targeted Adenovirus with the pfex System Ping Wu, Tarana A. Kudrolli, Wasim H. Chowdhury, Shawn E. Lupold, Ronald Rodriguez. 867 All-Trans Retinoic Acid (ATRA) Enhances Adenovirus Specific Integrin Receptor Expression on Dendritic Cells Airi Harui, Michael D. Roth, Saroj K. Basak. 868 Targeting of CRAd Agent to Human Squamous Cell Carcinomas of the Head and Neck (SCCHN) Zeng B. Zhu, Michael J. Mathis, Sharmila K. Makhija, Baogen Lu, Minghui Wang, Gene P. Siegal, David T. Curiel. 869 Adenovirus Vectors Targeted to HER3/4 Receptors for Breast Cancer Gene Therapy Sheena H. MacLeod, Mabrouk M. Elgadi, Gianluca Bossi, Kate C. Agopsowicz, Frank L. Graham, Mary M. Hitt. 870 Effect of Coagulation Factor X on the Infectivity of Adenoviruses Pseudotyped with Fibers from Subgroup D Alan L. Parker, John H. McVey, Simon N. Waddington, Suzanne M. K. Buckley, Jessica H. Doctor, Oscar Lopez-Franco, Menzo J. Havenga, Stuart A. Nicklin, Andrew H. Baker. 871 Characterization of Coagulation Factor IX Interactions with Human Adenovirus Fiber Knob Domains Using Tandem Mass Spectrometry and Surface Plasmon Resonance Oleksandr Kalyuzhniy, Catalin Doneanu, Martin Sadilek, Jan Seebacher, Dmitry M. Shayakhmetov. 872 Chimeric Adenoviral Vectors Displaying Fibers of Subgroup B Effectively Mediate Transgene Expression into Specific Cancer Cells Miho Murakami, Hideyo Ugai, Natalya Belousova, Masato Yamamoto, Maaike Everts, David T. Curiel. 873 Circumventing Induction of Neutralizing Antibodies to Adenovirus by Manipulating Vector Dose Marlene S. Strayer, Kristin Blauvelt, David S. Strayer. 874 Design and Characterization of Her2-Targeted Adenovirus Vectors Natalya Belousova, Marine Kojanyan, Galina Mikheeva, Victor Krasnykh. 875 Comprehensive Search for Factors Involved in the Innate Immune Response Induced by Adenovirus Vectors Haruna Sakurai, Katsuhide Igarashi, Katsuhisa Tashiro, Kenji Kawabata, Fuminori Sakurai, Shinnosuke Kurachi, Shinsaku Nakagawa, Ken-ichi Aisaki, Jun Kanno, Hiroyuki Mizuguchi. 876 Tropism Analysis of Several Primate-Derived Adenoviral Vectors in Leukemia Cell Lines Barbara Lombardo, Agostino Cirillo, Carmine Cozzolino, Barbara Izzo, Paola Rinaldi, Nicola Esposito, Paolo Morabito, Luigi Del Vecchio, Fabrizio Pane, Francesco Salvatore, Alfredo Nicosia, Riccardo Cortese, Lucio Pastore, Stefano Colloca. Adenovirus Vectors: New Technology 877 Extended Transgene Expression from a Non-Integrating Adenoviral Vector Containing Retroviral Elements Changyu Zheng, Joseph M. Vitolo, Weitian Zhang, Fumi Mineshiba, John A. Chiorini, Bruce J. Baum. 878 Genetic Incorporation of the Protein Transduction Domain of TAT into Ad5 Fiber Selectively Enhances Viral Infectivity Tie Han, Yizhe Tang, Hideyo Ugai, Juan L. Contreras, Hongju Wu. 108

110 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Adenovirus Vectors: New Technology continued 879 Improved Design of Fiber-Mosaic Adenoviruses Larisa Pereboeva, Svetlana Komarova, Justin Roth, Yuko Tsuruta, David T. Curiel. 880 Characterization of Potentially High Packaging Capacity Adenovirus Vector Deleting Minor Core Protein V Gene Hideyo Ugai, Anton V. Borovjagin, Long P. Le, Minghui Wang, David T. Curiel. 881 Hsmar2, a Newly Reconstructed Human Mariner Transposon Mediates Specific Gene Transfer in Human Cells of Expression Cassettes Flanked by TR-Hsmar2 Sequences Estel Gil, Ester Rodriguez, Lorena Ariza, Mireia Ferrer, Assumpcio Bosch, Olivier Danos, Miguel Chillon. 882 Fiber-Modified Adenovirus Vectors Containing the TAT Peptide in the Fiber Knob Have Efficient Gene Transfer Activity Shinnosuke Kurachi, Katsuhisa Tashiro, Haruna Sakurai, Fuminori Sakurai, Kenji Kawabata, Katsutoshi Yayama, Hiroshi Okamoto, Shinsaku Nakagawa, Hiroyuki Mizuguchi. 883 Localized Viral Gene Delivery through Co-Axial Electrospun Fibers I.-Chien Liao, Jason B. Liu, Kam W. Leong. 884 Adenovirus Complementing Cell Line for the Construction of Vectors with Inhibitory Transgenes Duncan McVey, Jason Gall, Chris Gunning, Michelle White, Damodar Ettyreddy, Chi Hsu, Amel Ahmed, Idalia Yabe, Alena Lizonova, Lauren Tetz, Douglas E. Brough. 885 Insulated Heat Shock Promoter Is Functional in Adenovirus Vectors, but Not in Oncolytic Adenoviruses Stanimira Rohmer, Astrid Mainka, Ilka Knippertz, Andrea Hesse, Dirk M. Nettelbeck. 886 Three pixs Can Be Incorporated into One Adenovirus Type 5 Virion Yizhe Tang, Long P. Le, Qiana L. Matthews, Tie Han, Hongju Wu, David T. Curiel. 887 Herpes Simplex Virus Type 1 Thymidine Kinase Sequence Fused to β-galactosidase Gene Increases Levels of β- Galactosidase Activity Per Genome of Both First Generation and High-Capacity Adenoviral Vectors In Vitro and In Vivo Mariana Puntel, Sonali Mondkar, A. K. M. Muhammad, Chunyan Liu, Sandra Sciascia, Weidong Xiong, Kurt M. Kroeger, Peter Czer, Donna Palmer, Philip Ng, Maria G. Castro, Pedro R. Lowenstein. 888 Application of Tetracyclin Inducible Promoter Based CRAd to Cell Vehicle System Koichi Takayama, Junji Uchino, Noriaki Nakagaki, Haiping Zhang, Ling Zhang, Akiko Harada, Taishi Harada, Yoichi Nakanishi. 889 VP16-Mediated Expression of Human Single-Chain Interleukin-12: Highly Efficient and Suppressible Gene Expression through Adenoviral Infection in Human Colon Cancer Andreas Block, Holger Wulff, Thorsten Krieger, Karen Krueger, Matthias Friesicke, Friedrich Thaiss, Hansjoerg Schaefer, Ansgar W. Lohse. 890 Capsid Protein IIIa as a Locale for Incorporation of Heterologous Peptides on Adenovirus Type 5 Matthew S. Beatty, Joel N. Glasgow, David T. Curiel. 891 Adenovirus 35 Vectors with Improved Transgene Capacity, and Ease of Production Duncan McVey, Mohammed Zuber, Damodar Ettyreddy, Idalia Yabe, Angela Bommersbach, Richard Grier, Douglas E. Brough, C. Richter King, Amel Ahmed, Megan Fultz, Andrew Glenn, Gary J. Nabel, Jason G. Gall. 892 A Novel Method for Adenovirus Purification and Titration Elina R. Makino, Chales M. Neville, Mikhail Kozlov. Non-Viral Delivery Strategies 893 Mechanism of Escape from Macropinosomes for Octaarginine Modified Nanoparticles Ayman El-Sayed, Ikramy A. Khalil, Kentaro Kogure, Hideyoshi Harashima. 894 Novel Approach for the Nuclear Delivery of Macromolecule Via Membrane Fusion in Non-Dividing Cell Asako Kudo, Hidetaka Akita, Arisa Minoura, Kentaro Kogure, Masaya Yamaguchi, Ikramy A. Khalil, Tomoya Masuda, Hideyoshi Harshima. 895 Polymer-DNA Formulations for Gene Transfer to Skeletal and Cardiac Muscles Caroline Roques, André Salmon, Marc Y. Fiszman, Elias Fattal, Yves Fromes. SATURDAY, JUNE 2 109

111 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Non-Viral Delivery Strategies continued 896 Development on Optimization of a Systemic Non-Viral Gene Delivery for Cancer with a Cleavable PEG System Hiroto Hatakeyama, Hidetaka Akita, Kentaro Kogure, Motoi Oishi, Yukio Nagasaki, Hideo Kobayashi, Hiroshi Kikuchi, Hideyoshi Harashima. 897 Octaarginine-Modified Multifunctional Envelope-Type Nano Device for Gene Delivery Kentaro Kogure, Ikramy A. Khalil, Shiroh Futaki, Susumu Hama, Hidetaka Akita, Masahisa Yamada, Yuji Mishina, Hideyoshi Harashima. 898 Skin Gene Therapy Using Chitosan Coated Liposomes Suna Ozbas-Turan, Julide Akbuga. 899 Improvement of Nuclear Transfer of pdna with Lipid Envelope-Typed Gene Delivery System by Utilization of Sugar as a Nuclear Targeting Ligand and Control of Lipid Stability Tomoya Masuda, Hidetaka Akita, Takashi Nishio, Kenichi Niikura, Kentaro Kogure, Asako Kudo, Kuniharu Ijiro, Hideyoshi Harashima. 900 Reducible Poly(Amido Ethylenetetramine) for Cardiac Gene Delivery James W. Yockman, Lane V. Christensen, David A. Bull, Zhiyuan Zhong, J. Feijen, SungWan Kim. 901 Non-Linear Pharmacodynamics in the Transfection Efficiency of a Non-Viral Gene Delivery System Rumiko Moriguchi, Kentaro Kogure, Hideyoshi Harashima. 902 Neural Retina Limits Delivery Non Viral Vectors to Retinal Pigment Epithelium Liesbeth Peeters, Niek N. Sanders, Stefaan C. De Smedt, Joseph Demeester. 903 Delivery of Biologically Active Nucleic Acid In Vivo Anne-Laure Bolcato-Bellemin, Marie-Elise Bonnet, Abdennaji Adib, Pascale Belguise, Jean-Paul Behr, Patrick Erbacher. 904 Adjusting Chitosan-DNA Nanoparticle by Characterizing the DNA Core and Chitosan Shelter Hui-jie Wang, Xingpin Qiu, Qin Shi, Francoise Winnik, Julio C. Fernandes. 905 Archaeosomes Based on Synthetic Tetraether-Like Lipids as Novel Versatile Gene Delivery Systems Tristan Montier, Gildas Rethoré, Tony Le Gall, Pascal Delepine, Sandrine Cammas-Marion, Pierre Lehn, Claude Ferec, Thierry Benvegnu. 906 Charge-Reversal Amphiphiles for DNA and sirna Transfection Carla A. Hernandez Prata, Sang Kwon, Dan Luo, Tom J. McIntosh, Mark W. Grinstaff. 907 srnavalet Transfection Reagent Carla Prata. 908 Peptidic Based Amphiphiles for Gene Delivery Carla A. Hernandez Prata, Sang Kwon, Dan Luo, Tom J. McIntosh, Mark W. Grinstaff. Physical Methods of Delivery II 909 In Vivo Gene Transfer to Duodenal Epithelial Cells Using a Chitosan-Based Vector Yvonne Luu, Anthony T. Cheung, Jun Gao, Kathryn Burton, Ada Arrango, Louis Huang, Eric C. Hsu. 910 Functional Rescue of the Rds Haploinsufficency Phenotype with Non-Viral Compacted DNA Nanoparticles Xue Cai, Steve J. Fiesler, Mark J. Cooper, Muna I. Naash. 911 Radiation-Guided Gene Delivery to Lung Tumor Xenografts by Annexcin V-Conjugated Liposomal Nanovector Ming Zhang, Nancy Xing, Theodore S. Lawrence. 912 Canine Erythropoietin as a Secreted Reporter System for the Study of Sleeping Beauty-Mediated Long-Term Expression in the Dog R. Scott McIvor, Erik R. Olson, Alice Hsu, Kerri Bostrom, Susan Sonntag, Kurt LaBresh, Jason Bell, Perry B. Hackett. 913 Aerosol Gene Delivery to the Lungs of Mice Guided by Magnetic Forces Petra Dames, Eugenia Lesina, Carsten Rudolph. 914 Gene Expression Profiles in Skeletal Muscle after DNA Electrotransfer Indicate a Highly Efficient and Safe Gene Therapy Platform John R. Zibert, Pernille H. Moller, Jens Eriksen, Julie Gehl. 110

112 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Physical Methods of Delivery II continued 915 Controlled Long-Lasting Hematocrit Increase by EPO Plasmid Electrotransfer Emmanuelle Fabre, Daniel Scherman, Pascal Bigey. 916 Muscle Characteristics Affect Plasmid Expression Following Electroporation in Both Large and Small Animals Patricia A. Brown, Amir S. Khan, Melissa A. Pope, Ruxandra Draghia- Akli. 917 Comparison of Electroporatively Delivered Non-Compacted and Compacted DNA Timothy G. Doody, John M. Piggott, Patrick F. Forde, Nicole Wilke, Anothony Morrissey, Declan M. Soden, Gerald C. O Sullivan. 918 Safety Aspects of Electro Gene Transfer (EGT) in Mouse Skeletal Muscle Christopher J. Mann, Xavier M. Anguela, David Callejas, Joel Montane, Lluis M. Mir, Fatima Bosch. 919 Development of SFV Based Gene Therapies for Use in Electroporative Cancer Treatment John M. Piggott, Declan M. Soden, Timothy G. Doody, Marina N. Fleeton, Gregory J. Atkins, Gerald C. O Sullivan. 920 Combined Electroporation and Ultrasound for Use in Chemotherapy Patrick F. Forde, John M. Piggott, Garrett Casey, Mark Tangey, Timothy G. Doddy, Declan M. Soden, Gerald C. O Sullivan. 921 Optimizing Ultrasound Conditions and Microbubble Concentration for Efficient Gene Transfer in Human Glioblastoma Cells Kadija Kaddur, Chantal Pichon, Truong A. Tran, Patrick Midoux, François Tranquart, Ayache Bouakaz. 922 Studies on Ultrasound-Mediated Transient Cell Membrane Permeabilisation (Sonoporation) Events In Vitro Ying Suet Li, Anthony P. McHale. 923 Enhanced Gene Transfer by Echo Contrast Agents: Comparison between BR1 and BR14 In Vitro and In Vivo Hitomi Endo, Katsuro Tachibana, Loreto B. Feril, Jr., Kazuki Yamaguchi. 924 Development of Minimally Invasive and Tissue Specific Gene Delivery with Novel Liposomal Bubbles Ryo Suzuki, Kaori Sawamura, Yusuke Oda, Tomoko Takizawa, Yoichi Negishi, Naoki Utoguchi, Kazuo Maruyama. Non-Viral Vectors: Biomaterials and Formulations 925 Engineered Modular Protein DNA-Carriers as Viral-Mimics for Intracellular Transport to the Cell Nucleus Dominic J. Glover, David A. Jans. 926 Non-Viral Gene Delivery from Templated Fibrin Tissue Engineering Scaffolds for Sustained Transgene Expression Justin M. Saul, Michael P. Linnes, Buddy D. Ratner, Cecilia M. Giachelli, Suzie H. Pun. 927 Controlled and Sustained Gene Release from Polymer DNA Matrices Scaffold for Heart Valve Tissue Engineering Wenzhong Li, Nan Ma, Karola Luetzow, Lee-lee Ong, Weiwei Wang, Andreas Liebold, Andreas Lendlein, Gustav Steinhoff. 928 Bioresponsive Endosomolytic Conjugates for DNA and sirna Delivery Martin Meyer, Arkadi Zintchenko, Alexander Philipp, Manfred Ogris, Christian Plank, Ernst Wagner. 929 New Series of Hydrophilic Polyhydroxylimine Transfection Agents Xiang Gao, Dexi Liu, Song Li. 930 Photochemical Control of Oligonucleotide Knockdown Activity Using Light Richard A. Blidner, Mindi H. Faubion, W. Todd Monroe. 931 Development of Bifunctional Polyethylene Glycol Gene Delivery Vehicles Nicole M. Moore, Tiffany R. Barbour, Shelly E. Sakiyama-Elbert. 932 Introduction of Imidazole to Chitosan for Enhanced Delivery of Nucleic Acids Bilal Ghosn, Huda Abdul-Razzak, Sudhir P. Kasturi, Krishnendu Roy. 933 In Vivo sirna Delivery Mediated by Dendritic Poly(L-Lysine) Ryohsuke Kurihara, Takahito Kawano, Takeshi Mori, Yoshiki Katayama, Takuro Niidome. SATURDAY, JUNE 2 111

113 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Non-Viral Vectors: Biomaterials and Formulations continued 934 Fully 2 -Deoxy-2 -Fluoro Substituted Nucleic Acids Induce RNA Interference in Mammalian Cell Culture Richard A. Blidner, Robert P. Hammer, W. Todd Monroe. 935 Brain Targeted Gene Therapy for Metachromatic Leukodystrophy: In Vitro Expression and Activity of a Melanotransferrin-Arylsulfatase A Fusion Protein Donna Y. Hou, Anna A. Li, Murray A. Potter. 936 In Vivo Efficient Gene Expression by Freeze-Dried DNA Complexes Coated by Polyanions Tomoko Ito, Keiko Abe, Naoko I. Tanaka, Yoshiyuki Koyama. 937 Transcriptional Enhancement by Synthetic Polyampholytes Mimicking HMG Protein Yoshiyuki Koyama, Chieko Yoshihara, Tomoko Ito, Kenichi Yoshikawa, Chwen Y. Shew. 938 Engineering Polypeptide-Coated Adenoviral Gene Delivery Vectors for Systemic Therapy of Prpstate Cancer Shuwen Koh, Lily Wu, Timothy J. Deming. 939 Pharmacogenomic Analysis of Non-Viral Gene Transfer: Feasibility of Effective Cell Differentiation toward Tissue Engineering Keiji Itaka, Kayo Masago, Kazunori Kataoka. 940 Efficient Delivery of Small Interfering RNA in Two Models: Vascular System in Vertebrate Embryo and Experimental Arthritis in Mouse Virginie Escriou, Maroun Khoury, Karine Bollerot, Florence Apparailly, Thierry Jaffredo, Daniel Scherman. 941 Intraperitoneal Administration of Calcium Phosphate Nanoparticles Encapsulating psvβgal Elicits Immune Response to Encoded Protein Amarnath Maitra, Savita Bisht, Dalia Chattopadhyay. 942 Ability of NT-Polyplex To Deliver a Suicide Gene to a Neuroblastoma Cell Line Hector A. Rubio-Zapata, Jesus D. Rembao-Bojorquez, Martha L. Arango- Rodriguez, Sandra Dupouy, Patricia Forgez, Daniel Martinez-Fong. 943 Recombinant High Mobility Group B-1 Peptide for Delivery of Nucleic Acids Kyunghwa Kim, Gwang Sig Yu, Joon Sig Choi, Minhyung Lee. 944 Formulation and Characteristation of Novel Cyclodextrin Gene Delivery Vectors Martin J. O Neill, Melanie Walsh, Colin P. Byrne, Raphael Darcy, Caitriona M. O Driscoll. 945 A Novel Chitosan-Based Polymeric Gene Carrier: Galactosylated 6-Amino-6-Deoxy-Chitosan Takeshi Nagasaki, Taku Satoh, Shinji Kakimoto, Seiji Shinkai. 946 Preparation of Novel Cationic Polydiacetylene Nanovesicles and Application to Gene Delivery In Vitro Gwang Sig Yu, Hye Choi, Yun Mi Bae, Jumi Kim, Jong-Man Kim, Joon Sig Choi. 947 Mechanically Enhanced Microencapsulated Cellular Gene Therapy Feng Shen, M. A. Jafar Mazumder, Nicholas Burke, Harald Stover, Murray Potter. 948 Long-Term Stability of Aqueous pdna/pei Complexes Graciela A. Nunez-Alonso, Lee A. Davies, Stephen C. Hyde, Deborah R. Gill. 949 Design of Gene Correction Devices for Improved Recognition by Protein Involved in Homologous Recombination Hiroyuki Kamiya, Hiroyuki Tsuchiya, Masayuki Uchiyama, Kazuhiro Hara, Hideo Inoue, Hideyoshi Harashima. Inborn Errors of Metabolism II Hemophilia Plus Others 950 Intraarticular Expression of Factor IX from Adeno- Associated Virus (AAV) Vectors Decreases Development of Hemophilic Joint Pathology Junjiang Sun, Narine Hakobyan, Leonard A. Valentino, Paul E. Monahan. 951 Novel AAV-FVIII Single Chain Vectors That Produce High Levels of FVIII Activity in Hemophilia A Mice Denise E. Sabatino, Amy M. Lange, Samuel J. Vidal, Rita Sarkar, Valder R. Arruda, Haig H. Kazazian, Jr. 112

114 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Inborn Errors of Metabolism II Hemophilia Plus Others continued 952 Long-Term FVIII Expression Via Sleeping Beauty in Liver Sinusoidal Endothelial Cells of Transgenic Mice Betsy T. Kren, Gretchen M. Unger, Alycia A. Trossen, Mark T. Reding, Robert P. Hebbel, Clifford J. Steer. 953 High-Level, Erythroid-Specific Human Factor IX Expression in Hematopoietic Chimeras Using Non-Myeloablative Conditioning? Alex H. Chang, Michel Sadelain. 954 Toward Hemophilia A Gene Therapy: Safety-Modified Gammaretroviral Vector Expressing a Secretion-Enhanced FVIII Molecule with Reduced Immunogenicity Ali Ramezani, Teresa S. Hawley, Robert G. Hawley. 955 An Essential Role of Factor VIII Light Chain in Facilitating Heavy Chain Secretion Lingxia Chen, Fuxiang Zhu, Juan Li, Hui Lu, Haiyan Jiang, Shangzhen Zhou, Rita Sarkar, Valder R. Arruda, Jinhui Wang, Chuma J. Chike-Obi, Hua Zhu, Glenn F. Pierce, Steven W. Pipe, Xiang-Qin Liu, Xiao Xiao, Rodney M. Camire, Weidong Xiao. 956 A High Specific Activity Factor VIII Variant and Its Application to AAV-Mediated Gene Transfer for Hemophilia A Yi-Lin Liu, Raffaella Toso, Heesoon Chang, Rodney Camire, Katherine High. 957 Factoring in ΦC31 Integrase as a Cure for Hemophilia Vanessa E. Gabrovsky, Christopher L. Chavez, W. Edward Jung, Annahita Keravala, Lauren E. Woodard, Michele P. Calos. 958 A Novel Approach to Proliferate Factor IX-Producing Hepatocytes Using Urokinase-Type Plasminogen Activator Transgenic SCID Mice for Establishment of Cell-Based Therapy of Hemophilia B Kohei Tatsumi, Kazuo Ohashi, Miho Kataoka, Chise Tateno, Katsutoshi Yoshizato, Michiyoshi Hisanaga, Hiromichi Kanehiro, Masaru Shibata, Midori Shima, Yoshiyuki Nakajima, Akira Yoshioka. 959 Specific Amino Acid Modifications To Increase Safety and Efficacy of Nonviral Gene Therapy of Factor VII for Hemophilia A with Inhibitors Steven C. Chen, Peiqing Ye, Amy D. Chen, Earl W. Davie, Carol H. Miao. 960 Lentiviral Modification of Liver Sinusoidal Endothelial Cells (LSEC) for Cell and Gene Therapy in Hemophilia A Antonia Follenzi, Sanj Raut, Louisa M. Faulkner, Rita Sarkar, Sanjeev Gupta. 961 Delivery of Human Coagulation Factor VIII Gene into Skeletal Muscle Cells Using Lentiviral Vector Hyun Jeong Jeon, Oak Hee Kim, Seung Taik Kim, Tae Keun Oh. 962 Liver-Restricted Administration of Lentiviral Vector during Isolated Hepatic Perfusion Results in Significant Reduction of Plasma Bilirubin Levels in the Gunn Rat Pascal van der Wegen, Gisela aan de Wiel-Ambagtsheer, Luc J. van der Laan, Timo L. ten Hagen, Bob J. Scholte. 963 Complete Phenotype Correction in Female PKU Mice by Repeated Administrations of Murine PAH cdna Using a Genome-Targeted phibt1 Bacteriophage Integration System Li Chen, Savio L. C. Woo. 964 Balloon Occlusion Catheter-Based Delivery of HDAd into the Nonhuman Primate Liver Results in Stable, High Level Transgene Expression with Minimal Toxicity Nicola Brunetti-Pierri, Gary Stapleton, Mark Law, Donna Palmer, Yu Zuo, Arthur Beaudet, Milton Finegold, Charles Mullins, Philip Ng. 965 High-Pressure Delivery Via Peripheral Vein Facilitates Transduction of Canine Liver with AAV Vectors Eduard Ayuso, Montane Joel, Anguela M. Xavier, Callejas David, Mann Chris, Garcia Felix, Andaluz Ana, Mingozzi Federico, High A. Katherin, Bosch Fatima. 966 Limited Correction of VLCAD Deficiency in Mice with a Native VLCAD Promoter David B. Schowalter, Tien V. Nguyen, Janice K. Daniels, J. Lawrence Merritt. 967 Non-Viral Gene Transfer into Gunn Rats for Prevention of Drug Toxicity and Investigation of UGT Isoenzyme Function In Vivo Istvan Danko, Zhen Jia. 968 Patent Inventorship of Principal Investigators in Phase I Gene Transfer Clinical Trials: An Empirical Analysis Karen L. Durell, Jonathan Kimmelman, Josephine Nalbantoglu, Richard Gold. SATURDAY, JUNE 2 113

115 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Cardiovascular Gene Therapy II 969 Paracrine Support Is a Predominant Mechanism by Which Pluripotent Adipose Stromal (Stem) Cells Promote Reperfusion of Ischemic Tissues Liying Cai, Brian H. Johnstone, Todd G. Cook, Zhong Liang, Dmitry Traktuev, Kenneth G. Cornetta, Elliot D. Rosen, Keith L. March. 970 Characterization, Isolation and Angiogenesis of Endothelial Cells Derived from Skeletal Muscle and Heart Nicholas Ieronimakis, Gayathri Balasundaram, Jeffrey S. Chamberlain, Morayma Reyes. 971 Development of High Throughput Functional Screening of Cardiovascular Therapeutic Genes Using the HVJ-E Vector and Identification of a Novel Angiogenic Peptide, AG-30 Tomoyuki Nishikawa, Hironori Nakagami, Akito Maeda, Yasuhiko Tabata, Katsuto Tamai, Yasufumi Kaneda. 972 P27kip1-Loaded Nanoparticle for the Inhibition of Intimal Hyperplasia in Mice Vein Grafting Model Jing Ynag, Cunxian Song, Xiaoou Lang. 973 Protective Effects of Limited In Vivo Gene Transfer of Phospholamban Antisense in a Catecholamine Induced Hypertrophic Rat Heart Model Motoki Sato, Peter O Gara, Stephen J. Fuller, Sian E. Harding. 974 Increased Collateral Development by Transplantation of Skeletal Myoblasts Infected with Adenoviral Vectors Expressing Hybrid Hypoxia-Inducible Factor 1α in a Rat Model of Hind Limb Ischemia Zhengyu Luo, Wei Zhang, Jeff Stewart, Hidetoshi Kajiwara, Akihiro Urabe, Adam Belanger, Karen Vincent, Geoff Akita, Seng H. Cheng, Canwen Jiang. 975 AP20187 Induces Insulin-Like Effects in Muscle and Liver of Diabetic Mice Transduced with AAV Gabriella Cotugno, Pietro Formisano, Ferdinando Giacco, Pasqualina Colella, Francesco Beguinot, Alberto Auricchio. 976 Additive Effect of AAV Mediated VEGF Gene and Mesenchymal Stem Cell Therapies on the Treatment of Ischemic Cardiomyopathy Hua Su, Yu Huang, Junya Takagawa, Jennifer Pons, Janice Arakawa- Hoyt, Jianqin Ye, William Grossman, Yuet W. Kan. 977 Optimization of Adeno-Associated Virus Mediated Apolipoprotein A-I Gene Expression in the Murine Liver Stefan F. C. Vaessen, Robert Jan Veldman, Elisabeth M. Comijn, Jolanda Snapper, Sanne Derks, Michael Hebert, Dirk Grimm, Mark A. Kay, Amit C. Nathwani, John J. P. Kastelein, Jaap Twisk, Jan Albert Kuivenhoven. 978 Superior Cardiac Transduction Efficacy Using a Pseudotyped AAV2/6 Vector Compared to a Heparin-Binding Site Deleted AAV2 in a Pig Model Corinna Lebherz, Philip Raake, Rabea Hinkel, Susanne Muller, Sebastian Delker, Christian Kupatt, Juergen A. Kleinschmidt, Oliver J. Mueller, Peter Boekstegers. 979 Chronic Expression of AZGP1 Following AAVrh.10-Mediated Gene Transfer Limits Weight Gain in Mice on High Fat Diets Neil R. Hackett, Jamie D. Schwartz, Holly Vanni, Ralf H. Hubner, Timothy P. O Connor, Ronald G. Crystal. 980 Delivery of VEGF Using AAV Vectors Promotes Functional Recovery of the Infarcted Myocardium by Sustaining Cardiomyocyte Contractility and Metabolism Lorena Zentilin, Silvia Camporesi, Lucia Pattarini, Serena Zacchigna, Uday Puligadda, Milena Sinigaglia, Mauro Giacca. 981 Rapid Purification by FACS of Myoblasts Expressing Specific VEGF Levels Ensures Controlled Angiogenesis In Vivo Heidi Misteli, Philipp Fueglistaler, Thomas Wolff, Roberto Gianni- Barrera, Lorenz Gurke, Michael Heberer, Andrea Banfi. 982 Self-Complementary raav Vector for Antioxidant Gene Delivery in Mouse Liver Ischemia-Reperfusion Injury Protection Lucia E. Rosas, Ian N. Hines, Michael D. Wheeler, Douglas M. McCarty. 983 Rapid and Widespread GFP Expression in Murine Myocardium Using scaav Lourdes M. Andino, Thomas Conlon, Stacy Porvasnik, Sanford Boye, William W. Hauswirth, Alfred S. Lewin. 114

116 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Neurologic II 984 Intrabody-Mediated Redistribution of Mutant Huntingtin as Gene Therapy Julie McLear, Erik Kvam, Nikolas Webster, Anne Messer. 985 Modulating SMN2 Pre-mRNA Splicing: Identification of Optimal Targets for Bi-Functional RNAs Travis D. Baughan, Monir Shababi, Christian L. Lorson. 986 Herpes Simplex Virus (HSV) Vector-Mediated Delivery of Glial Cell Line Derived Neurotrophic Factor (GDNF) and the GDNF Ligand Neurturin Rescues Erectile Dysfunction Following Cavernous Nerve Injury Ryuichi Kato, Darren P. Wolfe, Christian H. Coyle, Shaohua Huang, James B. Wechuck, William F. Goins, Fernando de Miguel, Taiji Tsukamoto, Joel B. Nelson, Michael B. Chancellor, Naoki Yoshimura, Joseph C. Glorioso. 987 Effects of Promoter and Viral Dose on AAV Vector-Mediated Gene Expression, Efficacy and Survival in a Mouse Model of Niemann-Pick A Disease Jie Bu, James C. Dodge, Catherine R. O Riordan, Robin J. Ziegler, Antonius N. Song, Joseph W. Foley, Jonathan A. Fidler, Eric M. Roskelley, Edward H. Schuchman, Lamya S. Shihabuddin, Seng H. Cheng, Marco A. Passini. 988 Antioxidant Enzymes Delivered Using SV40 Vectors Protect Primary Neurons In Vitro from Apoptosis Mediated by Transduced HIV-1 gp120 Lokesh Agrawal, J.-P. Louboutin, Beverly A. S. Reyes, Elisabeth Van Bockstaele, David S. Strayer. 989 Integration-Deficient Lentiviral Vectors Mediate Efficient Gene Transfer to Nervous System and Muscle Rafael J. Yáñez-Muñoz, Edmund Foster, Ping Yip, Stephen B. McMahon, Simon Waddington, Michael Antoniou. 990 Neonatal Intraperitoneal or Intravenous raav8 Injections for Targeting Lower Motor Neurons Kevin D. Foust, Amy Poirier, Christina A. Pacak, Terence R. Flotte. 991 Retrograde Axonal Transport of AAV Vectors Allows Extended Restoration of Dystrophin Expression by Exon Skipping in mdx Mice Brain Elise Peltekian, Ros Carine, Carole Gruszczynski, Cyrille Vaillend, Serge Laroche, Luis Garcia, Olivier Danos. 992 Ultrasound-Mediated Gene Transfer of HGF Improved Abeta- Induced Cognitive Impairment in Mice Associated with Upregulation of BDNF, Recovery of and Downregulation of Oxidative Stress Daisuke Takeuchi, Naoyuki Sato, Hitomi Kurinami, Munehisa Shimamura, Shuko Takeda, Mitsuru Shinohara, Toshio Ogihara, Ryuichi Morishita. 993 Gene Delivery and Biodistribution in the Brain Following Adeno-Associated Virus Serotype 5 Vector-Mediated Gene Transfer Lalitha Belur, Zenhong Nan, Zachary DeMorest, Chester B. Whitley, Walter Low, Carolyn Fairbanks, R. Scott McIvor. 994 Neural Expression of G Protein-Coupled Receptors GPR3, GPR6, and GPR12 Upregulates Cyclic AMP Levels and Promotes Neurite Outgrowth Shigeru Tanaka, Ken Ishii, Kazue Kasai, Sung Ok Yoon, Yoshinaga Saeki. 995 Enhanced Gene Transfer to Photoreceptors by Adenovirus Vectors Deleted in RGD Penton Application to the Rescue of Photoreceptor Degeneration in Two Models of Retinitis Pigmentosa Siobhan Cashman, Daly Gerard, William Brunken, Rajendra Kumar- Singh. 996 Intracerebral Delivery of AAV-2/5 Vectors To Treat Canine MPS IIIB N. Matthew Ellinwood, Karen L. Kline, Marie-Anne Colle, Elizabeth M. Snella, Jackie K. Jens, Song Lui, Yan Cherel, Jean Michel Heard. 997 Disseminated Gene Transfer to the CNS by Intraventricular Injection with Mannitol Pretreatment Using SV40-Derived Vectors Delivers Generalized Transgene Expression throughout the Brain Jean-Pierre Louboutin, Lokesh Agrawal, Beverly A. S. Reyes, Elisabeth J. van Bockstaele, David S. Strayer. 998 Simultaneous Silencing of Vanilloid Receptor TRPV1 and Pain-Related Cation-Channel P2X3 with a Single Lentiviral Vector Reduces Neuropathic Pain In Vivo Chung-Ren Lin, Kuan-Hung Chen, Yi-Shen Chen, Chien-Te Lee, Chien- Cheug Liu. SATURDAY, JUNE 2 115

117 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Neurologic II continued 999 AP20187-Mediated Activation of Ret Signalling as Regulated System for Neuroprotection in the Retina Mariacarmela Allocca, Umberto Di Vicino, Marco Petrillo, Francesca Carlomagno, Luciano Domenici, Alberto Auricchio Nerve-Directed Enkephalin Expression for Pain Therapy Report of Preclinical Safety and Efficacy Studies Darren Wolfe, David Krisky, James Wechuck, James Goss, Shaohua Huang, Joseph Glorioso Efficient Synapsin-dsRed and Synapsin-EGFP Labeling of Rat Neuronal Precursors and Human Postmitotic NT Neurons for Grafting in Rats with Spinal Ischemia-Induced Paraplegia Atsushi Miyanohara, Osamu Kakinohana, Silvia Marsala, Michael P. Hefferan, Tetsuya Fuchigami, Martin Marsala Adeno-Associated Virus Gene Therapy of Feline Gangliosidosis Douglas R. Martin, M. Begona Cachon-Gonzalez, Miguel Sena-Esteves, Nancy R. Cox, Timothy M. Cox, Henry J. Baker Glaucoma Gene Therapy: COX-2 and the Prostaglandin F Receptor Combined Reduce Intraocular Pressure in the Cat Eye Roman A. Barraza, Jay W. McLaren, Eric M. Poeschla. Lung and Respiratory Disease 1004 Repeated Administration of Lentiviral Vector to Mouse Nasal Epithelia Enhances Expression without Emergence of Blocking Mucosal Immune Responses Patrick L. Sinn, Ariadna C. Arias, Paul B. McCray, Jr Lifetime Airway Gene Expression from a Single Lentivirus Dose? Alice G. Stocker, Don S. Anson, David W. Parsons Electroporation-Mediated Gene Transfer of the β2- Adrenergic Receptor Reduces Bronchoconstriction in a Mouse Asthma Model James E. Norton, Tomasz J. Kuzniar, Gokhan M. Mutlu, Aaron F. Zirk, David A. Dean Development of an Integrase Deficient Lentiviral Vector for Transient shrna Expression in Respiratory Epithelia Marisa B. Banasik, Anthony J. Fischer, Paul B. McCray, Jr Influence of the Human and Murine CMV Enhancer on the Duration of Expression from CpG-Free pdna Vectors in the Mouse Lung Anne-Marie Green, Ian A. Pringle, Anna E. Lawton, Graciela A. Nunez- Alonso, Lee A. Davies, Nelson S. Yew, Seng H. Cheng, Deborah R. Gill, Stephen C. Hyde Expression of the Cystic Fibrosis Transmembrane Regulator (CFTR) Protein Following Recombination between Two AAV Vectors Christine L. Halbert, Siu-ling Lam, A. D. Miller Evidence of Progenitor Cell Transduction in Mouse Nasal Epithelium with Lentiviral Vector Pseudotyped with Sendai Virus Envelopes Katsuyuki Mitomo, Makoto Inoue, Hitoshi Iwasaki, Toshiaki Tabata, Mariko Yoshizaki, Noriko Nagashima, Kentaro Washizawa, Satoshi Fujikawa, Eiji Akiba, Duncan M. Geddes, Uta Griesenbach, Eric W. F. W. Alton, Mamoru Hasegawa Evaluation of Host Immune Response as a Barrier To Repeat Intranasal Administration of Lentivectors Angela M. Giddings, Manij Patel, John C. Olsen Non-Invasive Imaging of Airways and Other Organs at High Resolution in Mice David Parsons, Karen Siu, Ivan Williams, Richard Boucher, Kentaro Uesugi, Naoto Yagi The Route of Luciferin Administration Is a Critical Determinant of Detection of Pulmonary and Nasal Luciferase Expression by In Vivo Bioimaging Suzanne M. K. Buckley, Steven J. Howe, Adrian J. Thrasher, Andrew H. Baker, Tristan R. McKay, Simon N. Waddington Lentiviral Vector-Mediated Expression and Activity of Small Hairpin RNA Interference for the Epithelial Sodium Channel in Functional Models of Human Airway Epithelium Elena Copreni, Lucia Palmieri, Stefano Castellani, Francesca Tilesi, Fiorentina Ascenzioni, Massimo Conese CpG-Dependent Inflammatory Response after Delivery of Lipid/pDNA Complexes to Murine Lungs Reto P. Bazzani, Anusha Varathalingam, Nilu Goonetilleke, Seng H. Cheng, Ian A. Pringle, Stephen C. Hyde, Deborah R. Gill. 116

118 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Lung and Respiratory Disease continued 1016 Rabbit Model for Airway Gene Delivery of Helper-Dependent Adenoviral Vectors Using Novel Aerosol Formulations Huibi Cao, Kitty Leung, Xiaofei Li, Marvin Estrada, Helena Frndova, Rahul Kushwah, Benjamin H. Lee, Donna Palmer, Philip Ng, Peter Cox, Allan L. Coates, Jim Hu In Vivo Imaging of Cationic Lipid 67-Mediated Gene Transfer in Mouse Lung Uta Griesenbach, Cuixiang Meng, Raymond Farley, Lee A. Davies, Deborah R. Gill, Stephen C. Hyde, Makoto Inoue, Seng H. Cheng, Ronald K. Scheule, Mamoru Hasegawa, Gad Frankel, Siouxsie Wiles, Eric E. W. F. Alton Generation of a CpG-Free Clinical Trial Plasmid for Cystic Fibrosis Lung Gene Therapy Ian A. Pringle, Anna E. Lawton, Anne-Marie Green, Anusha Varathalingam, Rebecca L. Coles, Hazel Painter, Lee A. Davies, Graciela Nunez-Alonso, Seng H. Cheng, Deborah G. Gill, Stephen C. Hyde. Oligonucleotide Therapies II 1019 Using Comparative Proteomics as a Tool for Target Selection and Validation for Personalized RNAi Therapy John S. Vorhies, Neil Senzer, John J. Nemunaitis Why Simple Cationic Liposome Formulations Fail To Deliver sirna Efficiently In Vivo Kevin Buyens, Stefaan C. De Smedt, Joseph Demeester, Niek N. Sanders Highly Effective Bi-Functional shrnas for Cancer Gene Therapy Donald Rao, Padmasini Kumar, Viviana Mangual, Ila Oxendine, Chris Jay, Alex W. Tong, Phillip B. Maples, Neil Senzer, John J. Nemunaitis Delivery of a lacz shrna to Muscles of the ROSA26 Mouse Erin S. Kirkegaard, Leonard Meuse, Eric Finn, Joel R. Chamberlain Chitosan Oligomers as sirna Delivery Systems In Vitro Mohamed M. Issa, Sabina P. Strand, Kjell M. Vårum, Per Artursson Enhanced RNA Interference Using Reducible Poly(Amido Ethylenimine) Ji Hoon Jeong, Lane V. Christensen, James W. Yockman, Zhiyuan Zhong, Johan F. J. Engbersen, Won Jong Kim, Jan Feijen, Sung Wan Kim Silencing of Microphage Migration Inhibitory Factor (MIF) Via sirna Causes Attenuation of Growth and Induction of Apoptosis in Human Prostate Cancer Cells Nan Jiang, Hongwei Li, Yongxin Gao, Colin Sumners Gene Silencing for Epidermal Growth Factor Receptor Variant-III Induces Cell-Specific Cytotoxicity Shay E. Park, Vidya Bodempudi, Weihong Pan, Mary Jean Mauzy, Farnaz Yamoutpour, Tuba Esfandyari, Robert Kratzke, Arkadiusz Dudek, Donald M. O Rourke, Donald J. Tindall, Faris Farassati sirna Pool Targeting Different Sites of Human Hepatitis B Surface Antigen Efficiently Inhibits HBV Infection Yong Chen, Zhaoyang Ye, Ram I. Mahato Genetic Knock-Down of Human TNF-α by Lentivirus- Encoded shrnas Maria Jakobsen, Brian Moldt, Keld Kaltoft, Jacob G. Mikkelsen Specific and Efficient Regression of Hepatocarcinoma In Vivo by Liver-Specific Expression of htert-targeting Trans- Splicing Ribozyme Min-Sun Song, Joo-Han Lee, You-Sub Woo, Kyoung-Sook Cho, Jin Sook Jeong, In-Hoo Kim, Seong-Wook Lee Silencing of Macrophage Scavenger Receptor A and CD36 by RNA Interference Decreases Foam Cell Formation Jari P. Lappalainen, Petri I. Mäkinen, Suvi E. Koota, Jenni M. Huusko, Pia Leppänen, Jussi Aarnio, Mikko P. Turunen, Seppo Ylä-Herttuala Gene Expression and Silencing for Successful Islet Transplantation Feng Li, Ravikiran Panakanti, Ram I. Mahato Plasmid Based RNA Interference Controls Interleukin-1 Expression in Osteoarthritis Alan J. Nixon, Thomas H. Witte, Michael S. Scimeca Effect of Inhibition of CIITA by shrna on High Responder Rat Orthotopic Liver Transplantation Gang Liu, Ning Lu, Xianghui He, Na Zhao, Yujie Qiu, Liwei Zhu. SATURDAY, JUNE 2 117

119 Abstract Directory SATURDAY, JUNE 2 Saturday, June 2, 2007 Poster Session III continued 5:15 pm 7:00 pm Hall 4AB Stem Cell II 1034 Preliminary Result of Injection of Progenitors of Hepatocytes Derived of Mesenchymal Stem Cell as Salvage Treatment for End-Stage Liver Disease A Phase I/II Clinical Trial Pedram Kharaziha, Babak Noorinayer, Masoud Soleimani, Farivar Farzaneh, Narges Zali, Faramarz Derakhshan, Amir-Hooshang Mohammad Alizadeh, Homayoun Zojaji, Mohammad-Reza Zali Functional Characterization of Neural Stem Cells from a Murine Model of Globoid Cell Leukodystrophy: Relevance for the Development of Gene/Cell Therapy Approaches Margherita Neri, Maderna Claudio, Tononi Laura, Visigalli Ilaria, Biffi Alessandra, Marchesini Sergio, Naldini Luigi, Gritti Angela Effective Long-Term Transgene Expression by Self- Complementary Adeno-Associated Virus Serotypes to Human Mesenchymal Stem Cells Derived from Both Bone- Marrow and Umbilical-Cord Blood Sung Jin Kim, Won-Il Lee, Hwon Heo, Ohkyu Shin, Yunhee Kim Kwon, Heuiran Lee Foamy Virus Vectors for Gene Therapy in Human Embryonic Stem Cells Maja Z. Salva, Helen Gharwan, Carol B. Ware, Peirong Wang, Roli K. Hirata, James M. Allen, Erik Olson, David W. Russell Tissue Distribution of Human Multipotent Stromal Cells (hmscs) after Intravenous or Intracardiac Infusion into Mice Ryang Hwa Lee*, Min Jeong Seo*, Andrey A. Pulin*, Darwin J. Prockop Gene Therapy for Bone Tissue Engineering Using a Combination of 3-D Tissue Engineered Scaffold and Non- Viral Gene Carrier Hossein Hosseinkhani, Mohsen Hossienkhani, Ali Khademhosseini, Tony Azzam, Avi Domb Viral Vector-Mediated Expansion of Olfactory Ensheathing Glia for Spinal Cord Neuroregeneration Vega García-Escudero, María Jesús Martín-Bermejo, Ricardo Gargini, Maria Teresa Gallego-Hernández, Ana García, Diana Simón, Alberto Rábano, Javier Díaz-Nido, Jesús Ávila, Maria Teresa Moreno-Flores, Filip Lim Cell Density and Viral Vector Load Are Determinant Factors for Ex Vivo Lentiviral Transduction Efficiency of Hematopoietic Stem Cells Niek P. van Til, Carla J. L. Oerlemans-Bergs, Fatima S. F. Aerts-Kaya, Trudi P. Visser, Axel Schambach, Gerard Wagemaker, Monique M. A. Verstegen Human Adipocyte-Derived Stroma Cells Exert Potent Antitumorogenic Effects in Pancreatic Cancer Béatrice Cousin, Hubert Lulka, Mireille André, Jean-Pierre Chavouin, Jean-Marie Péron, Luc Pénicaud, Lucien Pradayrol, Louis Casteilla, Emmanuel Ravet, Louis Buscail, Pierre Cordelier Bone Marrow Stem Cell Gene Transfer as a Means To Deliver Genes to Injured Liver Alena A. Chekmasova, Jean-Pierre Louboutin, Mahender Singh, David S. Strayer Syngeneic Bone Marrow Transplant in a Murine Model of Mucopolysaccharidosis Type I Daniel A. Wolf, Lalitha R. Belur, Kelly M. Podetz-Pederson, Jennifer L. Gori, Walter C. Low, Chester B. Whitley, R. Scott McIvor Ex Vivo Transduction and Transplantation of Bone Marrow Cells for Liver Gene Delivery of alpha-1 Antitrypsin (AAT) Hong Li, Yuanqing Lu, Rafal P. Witek, Lung-Ji Chang, Martha Campbell-Thompson, Terence Flotte, Bryon Petersen, Sihong Song Non-Viral Genetic Modification of Stem Cells Melissa Hoare, Udo Greiser, Frank Barry, Timothy O Brien Evaluation of MIP-1α Effect on Transduction Rate of Gal-v Oncovirus on Cord Blood Stem Cell Kamran Alimoghaddam, Lili Moezzi, Mitra Khalili, Maryam Bahtar, Pantea Ghodis, Seyed Hamidolah Ghaffari, Alireza Arjmand, Ardeshir Ghavamzadeh Gene Expression in Differentiating Human ES Cells Shaochun Song, Chris Stabban, Jeanne Loring, Theodore Friedmann Human Multipotential Stromal Cells (MSCs) Repeatedly Create Their Own Niches as They Are Expanded and Re- Expanded from Single Cells into Colonies Joni H. Ylostalo, Nikolay A. Bazhanov, Darwin J. Prockop. 118

120 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Sunday, June 3, 2007 Oral Abstract Session 500 Clinical Gene Therapy 8:00 am 10:00 am Room 4C1/4C :00 AM Combined Modality Therapy of Locally Advanced Pancreas Cancer (LAPC) Integrating Radiation Inducible Gene Therapy with TNFerade : Preliminary Results of a Randomized Phase II/III Trial Neil Senzer :15 AM Regression of Glioblastoma Multiforme Following Intracranial Infusions of IL13-Zetakine + /HyTK + CTL Clones Michael C. Jensen, Sanjiv S. Gambhir, Shahriar S. Yaghoubi, Christine Wright, Araceli Naranjo, Jamie Wagner, Cherrilyn Bautista, Wen-Chung Chang, Brenda Williams, Behnam Badie :30 AM NV1020, a Genetically-Engineered Oncolytic Herpes Simplex Virus Type 1, for Human Colorectal Cancer Metastatic to the Liver J. J. Nemunaitis, R. Chari, Y. Fong, H. de Haan, M. Karrasch, T. Reid :45 AM A Phase 1/2 Clinical Study of Intra-Articular Administration of a Recombinant Adeno-Associated Vector Containing a TNF-α Antagonist Gene in Inflammatory Arthritis Philip J. Mease, Alison E. Heald, Pervin Anklesaria, 13G01 Study Group :00 AM Phase I Clinical Trials of Intramuscular Injection of raav2 and raav1-pseudotyped Versions of an Alpha-1 Antitrypsin (AAT) Vector in AAT-Deficient Adults Terence R. Flotte, Mark L. Brantly, Lindo T. Spencer, Margaret Humphries, Thomas J. Conlon, Carolyn T. Spencer, Amy Poirier, Wendy Garlington, Dawn Baker, Sihong Song, Ken I. Berns, Nick Muzyczka, Richard O. Snyder, Sue Washer, Barry J. Byrne :15 AM Infusion of Thymidine Kinase (TK) + Donor Lymphocytes Promotes a Rapid and Wide Immune Reconstitution and Permits Long-Term Survival after Hematopoietic Transplantation from a Haploidentical Donor C. Bonini, F. Ciceri, M. T. Lupo Stanghellini, A. Bondanza, Z. Magnani, S. Perna, M. Bernardi, J. Peccatori, P. Servida, F. Crippa, S. Kaneko, V. Valtolina, M. Ferrari, E. Provasi, M. Salomoni, L. Turchetto, S. Toma, K. Traversari, P. Bruzzi, L. Castagna, A. Santoro, J. Apperley, S. Slavin, S. Colombi, C. Gallo Stampino, M. Bregni, C. Bordignon :30 AM Intramuscular Administration of an AAV-1 Vector in Humans Results in Capsid-Specific T Cell Responses Federico Mingozzi, Janneke Meulenberg, Daniel Hui, Etiena Basner- Tschkarajan, Albertine de Jong, Petra Pos, Katherine High. Oral Abstract Session 501 RNA Viral Vectors: Genotoxicity and Integration 8:00 am 10:00 am Room :00 AM In Vivo Lentiviral SIN-LTR Vector Integration Is Nearly Random and Shows a Preponderance of Satellite DNA in Postmitotic Rodent Eyes Cynthia C. Bartholomae, Rafael J. Yanez-Munoz, Kamaljit S. Balaggan, Steven J. Howe, Angus MacNeil, Robin R. Ali, Adrian J. Thrasher, Christof von Kalle, Manfred Schmidt :15 AM The GTSG.org Database: A First Meta-Analysis of More Than Retroviral Gene Therapy Vector Insertion Sites Frank A. Giordano, Jens-Uwe Appelt, Marcel Zimmermann, Stephan Weinhardt, Agnes Wagenblatt, Heike Allgayer, W. Jens Zeller, Stefan Fruehauf, Stephanie Laufs :30 AM Hot Spots of Retroviral Integration in Human CD34 + Hematopoietic Cells Claudia Cattoglio, Giulia Facchini, Serenella Sartori, Antonella Antonelli, Annarita Miccio, Barbara Cassani, Manfred Schmidt, Christof von Kalle, Steve Howe, Adrian J. Thrasher, Alessandro Aiuti, Giuliana Ferrari, Alessandra Recchia, Fulvio Mavilio :45 AM Self-Inactivating Retroviral Vectors Harboring Cellular Promoters Reduce the Risk of Insertional Transactivation and Transformation Daniela Zychlinski, Axel Schambach, Ute Modlich, Johann Meyer, Sabine Knoess, Elke Grassman, Anjali Mishra, Christopher Baum :00 AM Validating Assays for Vector-Mediated Genotoxicity David W. Emery, Chang Long Li, George Stamatoyannopoulos :15 AM Modeling the Genotoxicity of Viral Vector Integration in a Tumor Prone Hematopoietic Stem Cell Transplantation Model Eugenio Montini, Daniela Cesana, Manfred Schmidt, Francesca Sanvito, Maurilio Ponzoni, Lucia Sergi Sergi, Fabrizio Benedicenti, Cynthia Bartholomae, Clelia Di Serio, Claudio Doglioni, Christof von Kalle, Luigi Naldini :30 AM HIV-Derived Lentivector Particles Persist in Hematopoietic Target Cells and Exhibit Delayed Release Kinetics Amy M. Skinner, Lee O Neill, Peter Kurre :45 AM Neonatal Retroviral Vector Gene Therapy Does Not Induce Tumors in Normal C57BL/6 Mice Xiucui Ma, Mindy Tittiger, Rachel Burrows, Lingfei Xu, Katherine P. Ponder. SUNDAY, JUNE 3 119

121 Abstract Directory SUNDAY, JUNE 3 Sunday, June 3, 2007 Oral Abstract Session 502 Synthetic Vehicles for In Vivo Applications 8:00 am 10:00 am Room 4C :00 AM A New Platform for Targeted sirna Delivery In Vivo: Dynamic Polyconjugates Darren H. Wakefield, David B. Rozema, So C. Wong, Jason J. Klein, David L. Lewis, Stephanie Bertin, Tom Reppen, Paula L. Roesch, James E. Wolff, Jon A. Wolff :15 AM Design and Small Animal Imaging of PEG-Stabilized DNA Nanoparticles Retargeted to the Liver Wenchoa Sun, Rachel Rosenblum, Zhenghong Lee, Mark Pagel, Pamela B. Davis, Assem G. Ziady :30 AM Aerosol Delivery of Concentrated pdna/pei Formulations to the Sheep Lung Lee A. Davies, Gerry McLachlan, Stephanie G. Sumner-Jones, David S. Collie, Stephen C. Hyde, Deborah R. Gill :45 AM Prolonged Gene Silencing in Hepatoma Cells and Primary Hepatocytes after sirna Delivery with Biodegradable Poly(β-Amino Ester)s Roosmarijn E. Vandenbroucke, Bruno G. De Geest, Stefaan C. De Smedt, Joseph Demeester, Niek N. Sanders :00 AM Non-Viral Targeted Gene Transfer to Liver Cells Wouter H. Driessen, Felix M. Kluxen, Benjamin M. Looney, Jason B. Bell, Perry B. Hackett, R. Scott McIvor, Sean M. Sullivan :15 AM Novel Cancer Cell-Specific Gene Expression Using PKCalpher-Responsive Peptide/Polymer Conjugate Jeong-Hun Kang, Daisuke Asai, Jun Oishi, Riki Toida, Moeko Ijuin, Byungdug Jun, Kim Jonghwan, Takeshi Mori, Takuro Niidome, Yoshiki Katayama :30 AM Target Specific Gene Delivery to Liver by a Novel Class of Bi-Functional Compounds Keun-Sik Kim, Yixiong Lei, Stolz Donna, Liu Dexi :45 AM Highly Efficient Gene Transfer with a Novel Degradable Pseudodendritic Oligoamine: An In Vitro and In Vivo Study Verena Russ, Manfred Ogris, Ernst Wagner. Oral Abstract Session 503 Gene and Cell Therapies for Muscular Dystrophies 8:00 am 10:00 am Room 4C :00 AM Distinct Transduction Profiles in the Dystrophic Dogs with raav Serotype 8 Sachiko Ohshima, Jin-Hong Shin, Akiyo Nishiyama, Katsutoshi Yuasa, Hiroyuki Nakai, Takashi Okada, Shin ichi Takeda :15 AM A Clinically Relevant Gene Therapy Approach for Duchenne Muscular Dystrophy by Vascular Delivery of Micro- Dystrophin Louise R. Rodino-Klapac, Paul M. Janssen, Chyrstal L. Montgomery, Ryan Jensen, Louis G. Chicoine, K. Reed Clark, Jerry R. Mendell :30 AM Prolonged AAV-Mediated Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy with a Brief Course of Immunosuppression Zejing Wang, Christian S. Kuhr, James M. Allen, Eric Finn, Paul Gregorevic, Jeffrey S. Chamberlain, Stephen J. Tapscott, Rainer F. Storb :45 AM Inhibition of Myostatin by Gene Therapy Increases Muscle Mass and Strength in a Mouse Model of Muscular Dystrophy Amanda M. Haidet, Liza Rizo, Chalonda R. Handy, Chris J. Shilling, Zarife Sahenk, Jerry R. Mendell, Brian K. Kaspar :00 AM AAV Vector-Mediated Expression of Follistatin Stimulates Skeletal Muscle Hypertrophy and Can Enhance Functional Performance in Models of Muscle Disease Paul Gregorevic, Norman A. Meznarich, James M. Allen, Miki Haraguchi, Leonard Meuse, Eric Finn, Jeffrey S. Chamberlain :15 AM Inhibiting Myostatin in Dystrophic Mice with Follistatin Improves the Success of Myoblast Transplantation Basma F. Benabdallah, Manaf Bouchentouf, Joel Rousseau, Pascal Bigey, Annick Michaud, Pierre Chapdelaine, Daniel Scherman, Jacques P. Tremblay :30 AM Vascular Endothelial Growth Factor Overexpression Improved Survival and Engraftment of Human Myoblasts into SCID Mouse Muscles Manaf Bouchentouf, Basma F. Benabdallah, Pascal Bigey, Jacques P. Tremblay :45 AM Transplantation of Autologous Genetically Modified Myoblasts: A Possible Treatment for Recessive Muscular Dystrophies Jacques P. Tremblay, Simon Quenneville, Pierre Chapdelaine, Zoé Coulombe, Christophe Pichavant. 120

122 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Directory Sunday, June 3, 2007 Oral Abstract Session 504 Vector and Transgene Immunology 8:00 am 10:00 am Room 606/ :00 AM A microrna-regulated Lentiviral Vector Can Mediate Stable Gene Transfer and Antigen-Specific Immunological Tolerance Following Systemic Administration Andrea Annoni, Brian D. Brown, Manuela Battaglia, Lucia Sergi Sergi, Luigi Naldini, Maria-Grazia Roncarolo :15 AM Adoptive Therapy of Tolerogenic Dendritic Cells Mediates Factor VIII Persistence in Hemophilia A Mice Michael P. Seiler, Vincenzo Cerullo, Christian Clarke, Viraj Mane, John Rodgers, Brendan Lee :30 AM Early Fetal Gene Delivery Induces Both Central and Peripheral Immune Tolerance Evan Colletti, Sean Lindtsedt, Paul Park, Esmail D. Zanjani, Graca Almeida-Porada, Christopher D. Porada :45 AM Modulating T Cell-Dependent Tolerance to the Transgene Product in Non-Human Primate Model of AAV-Mediated Liver Gene Transfer Federico Mingozzi, Nicole Hasbrouck, Etiena Basner-Tschkarajan, Shyrie Edmonson, Daniel Hui, Denise Sabatino, Shangzhen Zhou, Fraser Wright, Valder Arruda, Katherine High :00 AM Transgene Immunity Dictates the Stability of Efficient EGFP Gene Transfer by Conventional (ss) and Self Complementary (sc) AAV7 Vectors in Nonhuman Primate Liver Gaung-Ping Gao, Roberto Calcedo, Lili Wang, Lauren Mays, Rebecca Grant, Jim Miller, Tracie Irvin, James M. Wilson :15 AM Transient Blockade of ICOS Co-Stimulatory Pathways Induced Long-Term Tolerance to Factor VIII Following Nonviral Gene Transfer of Factor VIII into Hemophilia A Mice Baowei Peng, Peiqing Ye, Bruce R. Blazer, Hans D. Ochs, Carol H. Miao :30 AM Toll-Like Receptors Activation in the Innate and Adaptive Immune Response to Helper Dependent Adenoviral Vectors Vincenzo Cerullo, Michael Seiler, Daniel Wang, Viraj Mane, Christian Clarke, Brendan Lee :45 AM An In Vitro Model of CD8+ T Cell-Mediated Killing of AAV- Transduced Human Hepatocytes Etiena Basner-Tschakarjan, Daniel J. Hui, Shangzhen Zhou, Federico Mingozzi, Katherine A. High. Oral Abstract Session 505 Gene Expression in Animal Model Systems 8:00 am 10:00 am Room :00 AM Prevention of Type I Diabetes Recurrence Following Islet Transplantation in Diabetic NOD Mice by Gene Therapy Chaorui Tian, Mohammed Javeed Ansari, Jesus Paez-Cortez, Jessamyn Bagley, Mohamed Sayegh, John Iacomini :15 AM Induction of Molecular Chimerism in Rhesus Macaques Reconstituted with Autologous CD34+ Cells Transduced with the Gene Encoding the α(1-3)galactosyltranferase (αgt) Lorenzo Benatuil, Robert Donahue, Aylin Bonifacino, Cynthia E. Dunbar, John Iacomini :30 AM Long-Term Retrovirus Expression in Neural Stem Cells and Their Neuronal Progeny for MeCP2 Gene Therapy of Rett Syndrome Mojgan Rastegar, Peter Pasceri, Maisam Makarem, Shauna Elliot, Leanne Jamieson, Megumi Adachi, Frederick S. Jones, Mark Henkelman, Ian Clarke, Peter Dirks, James Ellis :45 AM Comparison of dsaav Serotypes for Therapeutic Gene Transfer to Islets for Abrogation of Autoimmunity in Diabetes Daniel F. Gaddy, Khaja K. Rehman, Xiao Xiao, Suzanne Bertera, Nick Giannoukakis, Paul D. Robbins :00 AM Biological Identification of Ankyrin-1 Promoters Transcribed at Higher Rates for Globin Gene Therapy Karina Laflamme, Laura Elnitski, Patrick G. Gallagher, David M. Bodine :15 AM Neuronal Apoptosis Related to HIV-1 gp120 Can Be Inhibited by Different Strategies Using rsv40-delivery of Antioxidant Enzymes Jean-Pierre Louboutin, Lokesh Agrawal, Beverly A. S. Reyes, Elisabeth J. van Bockstaele, David S. Strayer :30 AM Targeted Expression of BikDD Eradicates Pancreatic Tumors in Noninvasive Imaging Models Xiaoming Xie, Weiya Xia, Hsu-Ping Kuo, Zheng Li, Qingqing Ding, Su Zhang, Jing-Yu Lang, Paul L. Chiao, James L. Abbruzzese, Mien-Chie Hung :45 AM Transcriptionally Targeted Cancer Gene Therapy for Small Cell Lung Cancer Nina Pedersen, Thomas T. Poulsen, Camilla L. Christensen, Vagn N. Handlos, Michael S. Lan, Mary B. Breslin, Rajagopal Ramesh, Began Gopalan, Jack A. Roth, Hans S. Poulsen. SUNDAY, JUNE 3 121

123 Abstract Directory SUNDAY, JUNE 3 Sunday, June 3, 2007 Oral Abstract Session 506 Oligonucleotide Therapies II 8:00 am 10:00 am Room :00 AM Inhibition of HIV-1 Replication by Lentiviral Vectors Harboring Long Hairpin RNAs Masayuki Sano, Haitang Li, Mahito Nakanishi, John J. Rossi :15 AM Targeted Inactivation of the CCR5 Gene in Human Cells Using Peptide Nucleic Acids Ranjit S. Bindra, Erica B. Schleifman, Peter M. Glazer :30 AM Novel Cell Type-Specifical Aptamer-siRNA Delivery System for HIV-1 Therapy Jiehua Zhou, Haitang Li, John J. Rossi :45 AM Rational Design Leads to More Potent RNA Interference Targeting Hepatitis B Virus Kathy Keck, Anton P. McCaffrey :00 AM Novel Bifunctional shrna Knockdown Therapeutics to Individualized Cancer Targets John J. Nemunaitis, Phillip B. Maples, Donald Rao, Joseph Kuhn, Yuqiao Shen, Alex W. Tong, Chris Jay, Padmasini Kumar, Neil Senzer :15 AM Using Let-7 microrna To Inhibit Pancreatic Cancer Cells Proliferation Laurie Parmentier, Jérôme Torrisani, Barbara Bournet, Anny Souque, Michèle Bouisson, Jean Escourrou, P. Senesse, Marc Barthet, N. Lesavre, Philippe Ruszniewski, Dermot O Toole, A. Aubert, Pascal Hammel, Philippe Levy, Janick Selves, Lucien Pradayrol, Louis Buscail, Pierre Cordelier :30 AM Argonaute-2 Is a Key Limiting Factor for Therapeutic RNAi in the Liver Dirk Grimm, Lora Wang, Joyce S. Lee, Theresa A. Storm, Mark A. Kay :45 AM Prevention and Reversal of Autoimmune Diabetes by Microsphere-Formulated Antisense Oligonucleotiudes Targeting Costimulation Karen Nylander, Jo Harnaha, Robert Lakomy, Alexis Styche, Kimberly Gillis, Larry Brown, Massimo Trucco, Nick Giannoukakis. Oral Abstract Session 507 Stem Cells Basic Science Session 8:00 am 10:00 am Room 608/ :00 AM High Proteasome Activity Is a Common Feature among Stem Cells of Different Lineages and Restricts Lentiviral Gene Transfer Francesca R. Santoni de Sio, Paolo Cascio, Angela Gritti, Margherita Neri, Maurilio Sampaolesi, Silvia Colleoni, Cesare Galli, Luigi Naldini :15 AM Enhanced Levels of the Gata-2 Transcription Factor Result in Myeloid Expansion Coupled with Inhibition of Lymphoid Development in the Absence of Leukemia Kyle Johnson, Satish Nandakumar, Tamara I. Pestina, Stacy Throm, Derek A. Persons :30 AM Targeting p18 Ink4c for Ex Vivo Expansion of Human Hematopoietic Stem Cells Hui Yu, Hongmei Shen, Feng Xu, Xiaoxia Hu, Tao Cheng :45 AM Increased Proliferation and Self Renewal Capacity of CD34+ Sca-1+ Putative Stem Cells in the Lung by Adenovirus- Mediated Overexpression of Sonic Hedgehog Anja Krause, Ju Joh, Austen Gess, Ralf H. Hubner, Yaqin Xu, Howard Lou, Philip L. Leopold, Ronald G. Crystal, Stefan Worgall :00 AM Prospective Isolation of Multipotent Adult Stem Cells from Skeletal Muscle Gayathri Balasundaram, Nicholas Ieronimakis, Morayma Reyes :15 AM Attempt for Enrichment and Characterization of Ovarian Cancer Stem Cells Zong-Yi Li, Robert Strauss, Pavel Sova, Ying Liu, Nicole Urban, Charles Drescher, Andre Lieber :30 AM Bone Marrow Is an Essential Source of Keratinocytes in the Grafted Skin Katsuto Tamai, Takehiko Yamazaki, Satoru Otsuru, Takenao Chino, Yasushi Kikuchi, Keisuke Nimura, Takashi Shimbo, Yasufumi Kaneda :45 AM Inducible LRP5 (ilrp5) Recapitulates Role of LRP5 Dimerization in Wnt/b-Catenin Signaling and Stem Cell Expansion Dongsu Park, Payam Shahi, Mamatha Seethammagari, David M. Spencer. 122

124 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index 13G01 Study Group Aagard, Lars aan de Wiel-Ambagtsheer, Gisela Aarnio, Jussi Abbruzzese, James L Abdel Fattah, El Moataz Abdul-Razzak, Huda Abe, Keiko Abe, Satoshi Abe, Takayuki Abe, Tatsuya Abkowitz, Janis L Abraham, John M Abrescia, Chiara Acland, Gregory M Acosta, Andres Adachi, Megumi Adaka, Satomi Adamo, P. Filippo Adams, Marv Adams, Robert Ader, Marius Adib, Abdennaji Adriaansen, Janik Advexin H N Study Group...45 Aebischer, Patrick Aerts-Kaya, Fatima S. F Africa, Liza M Agadjanian, Hasmik Agarwal, Rachana Agbandje-McKenna, Mavis Agopsowicz, Kate C Agrawal, Lokesh...367, 526, 530, 532, 543, 559, 682, 988, 997, 1095 Agrawal, Nalini Aguilar, Brenda...581, 598 Aguilar-Cordova, Estuardo Ahlen, Gustaf N Ahmed, Amel...884, 891 Ahn, Cheol-Hee Ahn, Hae-Sook Ahn, Kwnag-soo Ahn, Kyuseon Ahn, Miwon Aird, William C Airenne, Kari J...307, 393, 464, 467, 468 Aisaki, Ken-ichi Aiuti, Alessandro...238, 247, 648, 766, 1060 Aizawa, Emi Akbuga, Julide Akbulut, Hakan Aker, Mari Akiba, Eiji Akita, Geoff Akita, Hidetaka...400, 704, 894, 896, 897, 899 Akitomo, Kimiyo Akkina, Ramesh...292, 399, 558, 563, 569, 572, 573 Akubulut, Hakan Alauddin, Mian Albelda, Steven Alber, Sean Alberti, Michael O...15 Aldrighetti, Luca...39 Alemany, Ramon Alessandra, Biffi Alexander, John J Alfaro, Maite Ali, Robin R Alimoghaddam, Kamran...291, 1047 Al-Khadra, Saba Allen, Cornell Allen, Cory...51 Allen, James...113, 127, 134, 409, 721, 771, 1037, 1076, 1078 Allgayer, Heike Allikmets, Rando Allo, Valerie Allocca, Mariacarmela...826, 999 Almeida-Porada, Graca...294, 1084 Almqvist, Per M...41 Altomonte, Jennifer...49 Alton, Eric , 1017 Alvarez, Ronald D...182, 440 Alvarez, Xavier...27 Alvarez-Lozano, Eduardo Alves, Paula M

125 Abstract Author Index Alwin, Stephen Amabile, Nicolas Amalfitano, Andrea...9, 11, 555, 659, 660, 664 Ambrosi, Alessandro Amende, Ivo Amiaud, Jerome Amoscato, Andrew Ampong, Beryl N An, Dong Sung Ana, Andaluz Anagnostopoulos, Achilles Anagnou, Nicholas P Anai, Satoshi...496, 685 Anan, Mitsuhiro Andersen, Peter R Anderson, Daniel G...43 Anderson, Jonathan...309, 312 Anderson, Joseph S...558, 563, 572, 573 Anderson, Karl Anderson, Mathew L Andersson, Helen...361, 587 Andino, Lourdes M Ando, Dale...373, 436, 585 Andolfi, Grazia...238, 766 André, Franck M André, Mireille Andreani, Marco Andresen, Thomas L Andrew, Bird Andrew, Susan E Andurkar, Shridhar V Aneja, Manish K Ang, Brian U Ang, John Angela, Gritti Anguela, Xavier M Anklesaria, Pervin Annoni, Andrea Ansari, Mohammed Javeed Anson, Donald S...68, 416, 1005 Anssi, Mahonen J Antonelli, Antonella Antoniou, Michael...250, 651, 989 Anwer, Khursheed...170, 182 Aoki, Kazunori Aoki, Motokuni Aoki, Noboru Apparailly, Florence Appelt, Jens-Uwe Apperley, J Appledorn, Daniel...9, 11, 659, 660, 664 Arakawa-Hoyt, Janice Aramaki, Yukihiko Arango-Rodriguez, Martha L Arap, Wadih Aravindaram, Kandan...156, 164 Arbuthnot, Patrick...363, 707 Ardiani, Andressa Arditi, Moshe Argyros, Orestis Arias, Ariadna C Ariza, Lorena Arjmand, Alireza Armeanu, Sorin Armendariz-Borunda, Juan...449, 452, 455 Armentano, Donna Arnett, Andrea L. H Arnould, Sylvain Aronovich, Elena L...109, 339 Aronow, Bruce J Arrango, Ada Arruda, Valder...2, 745, 843, 951, 955, 1085 Arsic, Nikola Artursson, Per Arya, Suresh K Arzaga, Rosemarie Asada, Kazunori Asahi, Saori Asai, Daisuke Asai, Tomohiro...231, 710 Asari, Sayaka Ascenzioni, Fiorentina Ashar, Jignya Ashe, Karen

126 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Aslanidi, George...102, 829 Asokan, Aravind Astone, Dalila...13 Ataai, Mohammad M Athanasiou, Evangelia Atkins, Gregory J Aubert, A Aubert, Howard...44 Aubourg, Patrick Auclair, Christian...73 Aucoin, Marc G...89 Audit, Muriel Aurelian, Laure...459, 472 Auricchio, Alberto...8, 104, 112, 746, 826, 975, 999 Ausubel, Lara J Ávila, Jesús...535, 1040 Aviram, Michael Awedikian, Rafi...98 Ayesh, Suhail Ayuso, Eduard...120, 965 Azzam, Tony Baad, Michael Baatz, John E Backhouse, Jo...8 Backus, Carey Badie, Behnam Badr, Christian E Bae, Yun Mi Baek, Hyunjung Baekelandt, Veerle Bagheri, Mohamad Bagley, Jessamyn Bahtar, Maryam Baigen, Zhang Baila, Stefano...2 Bailey, Jeff...246, 652 Bailey, Susannah Baird, William H...394, 608 Bajgelman, Marcio C Baker, Alison Baker, Andrew H...329, 330, 355, 619, 857, 870, 1013 Baker, Dawn Baker, Eilidh Baker, Henry J...727, 1002 Baker, Kate...27 Baker, Kim Bakker, Andrew...85, 86, 88, 348 Balaggan, Kamaljit S Balasundaram, Gayathri...139, 970, 1110 Balimane, Praveen Ballabio, Andrea...94, 746 Ballinger, Robert A Ballmaier, Matthias Baltimore, David Banasik, Marisa B Bandyopadhyay, Sarmistha...827, 852 Banfi, Andrea...117, 981 Bangari, Dinesh S Bangtsson, Mia Bankiewicz, Krystof...435, 724 Banks, Glen...127, 134, 409 Baodong, Sun Baran, Szczepan W Baranick, Brian T Barbour, Tiffany R Barcia, Carlos...34, 176, 181, 538 Bargouti, Maggie Barichello, Jose M...493, 710 Barichievy, Samantha Barlow, Robert Barnes, Justin A Barnes, Mack N Barraza, Roman A Barrera, Jose Barrett, Alan D. T Barry, Frank...354, 1046 Barthet, Marc Bartholomae, Cynthia , 1063 Bartido, Shirley Bartlett, Jeffrey S...77, 78, 383, 725 Bartoli, Marc Bartsevich, Victor Bartus, Raymond T Barve, Minal...188, 443,

127 Abstract Author Index Basak, Saroj K Bashtar, Maryam Basner-Tschkarajan, Etiena...259, 1056, 1085, 1089 Bateman, Raynard L Batsis, Ioannis Battaglia, Manuela Bauche, Cecile...73 Bauer, Jr, Thomas R...243, 771 Bauerschmitz, Gerd...216, 610, 617, 619 Baughan, Travis D Baum, Bruce...286, 318, 403, 628, 671, 841, 877 Baum, Christopher...246, 327, 647, 770, 805, 1061 Baumhover, Nicholas J Bautista, Cherrilyn Bauzon, Maxine...221, 285 Baxter, Sarah Bayer, Matthew A Bayo-Puxan, Neus Bazhanov, Nikolay A Bazzani, Reto P Beall, Clifford J...84, 159, 823 Beard, Brian C...66, 239, 323, 378, 424, 562, 772, 800 Beattie, Craig W Beattie, Stuart G Beatty, Matthew S Beauchesne, Pascal R...817, 818 Beaudet, Arthur...417, 964 Beausejour, Christian Bedrosian, Jeff Beebe, Kate...66, 800 Beer, Shelly Beguinot, Francesco Behr, Jean-Paul Bekradda, M...45 Belanger, Adam...353, 974 Belguise, Pascale Bell, Christie Bell, Jason...24, 109, 339, 482, 912, 1070 Bell, Peter...258, 661, 743 Bellu, Anna Rita...230, 624 Belousova, Natalya...437, 872, 874 Belur, Lalitha...109, 220, 686, 993, 1044 Ben, Yong...50 Benabdallah, Basma F , 1080 Benatuil, Lorenzo Benedicenti, Fabrizio...303, 1063 Benihoud, Karim Bennett, Jean...525, 743, 745, 819, 854 Bennicelli, Jeannette...432, 745 Benrashid, Ehsan...128, 701 Bentsion, Dmitry Benvegnu, Thierry Benvéniste, Olivier Benza, Raymond L Bercury, Scott D Berendzen, Kristen Bergan, Lindsay Bergen, Jamie M Bergenheim, Tommy A Berger, Carolina Berges, Bradford K Bernardi, M Bernd, Hauck Berns, Ken I Berns, Kenneth I Berraondo, Pedro...362, 831 Bertera, Suzanne Bertin, Stephanie Bertrand, Lise Bertsch, Uwe Bet, Anne Beutel, Abram Bhagavan, Hemmi N Bhakta, Vipul Bhasker, C. Ramana Biasco, Luca...238, 766 Bier-Laning, C...45 Biesmans, Dennis...88 Biffi, Alessandra...303, 350, 423 Bigey, Pascal...915, 1079, 1080 Bilney, Adam Bindra, Ranjit S Biniamin, Shalva Binley, Katie

128 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Biral, Erika Bird, Andrew...352, 414 Birman, Tatiana Bishop, Kathie M Bisht, Savita Bitzer, Michael Black, Margaret E...198, 642 Blanchard, Stéphane...29 Blanks, Janet C Blau, Helen M Blauvelt, Kristin Blazer, Bruce R...95, 1087 Bleiziffer, Oliver Blidner, Richard A...930, 934 Block, Andreas Blokker, Juliette C Blondeau, Johanna Bochner, Bernard H Bockstaele, Elisabeth Van...543, 988 Bodempudi, Vidya Boden, Scott D Bodine, David M Boekstegers, Peter Bogan, Dan Bogan, Janet Bogdanovich, Sasha Bohl, Delphine...29 Bolcato-Bellemin, Anne-Laure Bollard, Catherine M Bollerot, Karine Bolund, Lars Bommersbach, Angela Bomstyk, Karol Bondanza, A Bondanza, Attilio...39 Bondioli, Kenneth...17 Bonetti, Ciro Bonifacino, Aylin...241, 1091 Bonini, C Bonini, Chiara...39 Bonnet, Marie-Elise Bordignon, C Bordignon, Claudio...37, 39, 238, 350, 766 Borges, Katharine Borovjagin, Anton V...440, 727, 880 Borquez-Ojeda, Oriana Bosch, Assumpcio...864, 881 Bosch, Fatima Bossi, Gianluca Bosticardo, Marita Bostick, Brian P...133, 850 Bostrom, Kerri Both, Gerald W Bouakaz, Ayache Boucas, Jorge...75 Bouchentouf, Manaf , 1080 Boucher, Richard Boudreau, Ryan L Boudreaux, Mary K Bougiouklis, Dimitris Bouisson, Michèle Boulanger, Pierre Boulikas, Teni Boulis, Nicholas M...77, 284, 531, 677 Bourassa, Erick Bourgeois, Jacqueline Bournet, Barbara...204, 1103 Bowen, Bruce D...817, 818 Boweres, William J Bowers, William J...30, 458, 539 Bowles, Dawn E...118, 390 Boye, Sanford...433, 983 Boye, Shannon E Boyer, Jean D...280, 751 Boyer, Julie L...158, 419, 582, 597, 655, 752, 755 Brady, James P Brake, David Brakenhoff, Ruud H Bramson, Jonathan L Brans, Richard Brantly, Mark L Brasky, Kathy...13 Bratescu, Laura Braun, Serge

129 Abstract Author Index Braun, Stephen E Braun, Werner Brayman, Andrew A...20 Brazelton, Tim Breakefield, Xandra O Bregni, M Bregni, Marco...37 Breitbart, Eyal Bremer, Edwin...46 Brenner, Malcolm K...38, 368, 375 Brenner, Sebastian Brentjens, Renier Brentjens, Renier J Breous, Ekaterina Breslin, Mary B Bridgeman, John S Brigida, Immacolata Brilliant, Murray H Bringas, John Brocker, Thomas Broderick, Kate Brody, Joshua Brondani, Vincent Brooke, Nicola Brooks, Alan Brough, Douglas E...439, 753, 860, 884, 891 Broun, Emily Brown, Brian D...405, 434, 774, 1082 Brown, Christine E...581, 594, 598, 663, 780 Brown, Eric J Brown, Larry Brown, Matthew Brown, Patricia A...17, 916 Brown, Talmage T Bruder, Joseph T...16, 148, 153 Brunetti-Pierri, Nicola...417, 964 Brunhoeber, Elaine Brunken, William Brunt, Elizabeth M...1 Bruscia, E...64 Bruzzi, P Bryan, Caroline Brzezinski, Margaret...105, 106 Bu, Jie Büchel, Janine Buchholz, Christian J...218, 813 Buchlis, George Buchsbaum, Donald J Buckler, Joshua M Buckley, Suzanne M. K...324, 330, 416, 870, 1013 Bueno-Topete, Miriam Ruth M. R Bull, David A Buning, Hildegard...75 Bunnell, Bruce A...27, 719 Bunte, Ralph...2 Burdorf, Lars Burger, Corinna Burgueno, Joan F Burke, Nicholas Burkholder, Tanya H Burnight, Erin R Burris, Clark A Burroughs, Kevin D Burrows, Rachel Bursill, Christine A...23 Burstein, Haim...57 Burton, Jeremy...233, 627 Burton, Kathryn...346, 909 Buscail, Louis...204, 633, 1042, 1103 Busik, Julia V Buyens, Kevin Byers, Peter H Byrne, Barry J...821, 1054 Byrne, Colin P Byrne, Melissa J Byrnes, Andrew P...12, 856 Byun, Jonghoe Cabaniols, Jean-Pierre Cachon-Gonzalez, M. Begona Cai, Liying Cai, Xue Caizergues, Didier Calarota, Sandra A Calcedo, Roberto...8, 155, 255, 257, 258, 366, 743,

130 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Caldwell, Scarlett M Callejas, David Calos, Michele P...337, 346, 542, 957 Cambois, Gilles Camenisch, Todd D Camire, Rodney...955, 956 Cammas-Marion, Sandrine Campbell, Jacquelyn A Campbell-Thompson, Martha...110, 825, 1045 Campo, Joseph J...148, 153 Campochiaro, Peter A Camporesi, Silvia Campos, Ruy R Cancelas, Jose A...394, 608 Candolfi, M...178, 758 Candolfi, Marianela...34, 176, 177, 181, 252 Candotti, Fabio Cano, Marisol D Cantin, Leo Cantore, Alessio...405, 774 Cao, Huibi Capacio, Vanessa Capotondo, Alessia...303, 350, 423 Carbonaro, Denise Carey, Michael Carillo, Bruno Carine, Ros Carlier, Pierre Carlomagno, Francesca Carlton, Erin...284, 531, 677 Carmo, Marlene Carner, Kristin Carnes, Aaron Carnish, Erin R Carrer, Alessandro Carrondo, Manuel J. T Carter, Barrie...57 Carter, Mark S Cartier, Nathalie Caruso, Manuel...209, 430 Carvalho, Leonardo P Casales, Erkuden Cascio, Michael...28 Cascio, Paolo Case, Casey C Casey, Ann E Casey, Garrett Cashman, Siobhan...709, 995 Cassani, Barbara...238, 766, 1060 Castagna, L Casteilla, Louis...633, 1042 Castellani, Stefano Castro, M. G Castro, Maria G...34, 174, 176, 177, 181, 252, 538, 758, 887 Castro-Govea, Yanko Cathomen, Toni...288, 781 Catron, Daniel J Cattaneo, Roberto...218, 750, 759, 808, 813 Cattoglio, Claudia Cavallo, Federica Cawley, Naimh X Cawley, Niamh X Cazzola, Matteo Cearley, Cassia N Cease, Kemp Cebotaru, Liudmila Cechin, Sirlene Cemazar, Maja Cerullo, Vincenzo...406, 1083, 1088 Cervantes-Kardasch, Victor H Cervelli, Tiziana...6 Cesana, Daniela Cesani, Martina...96, 303, 350 Cesari, Rossano...250, 651 Chada, Sunil...197, 380 Chadderton, Naomi Chae, Jin-A Chakrabarty, Ananda M Chamberlain, Jeffrey...113, 126, 127, 129, 134, 139, 409,...721, 970, 1076, 1078 Chamberlain, Joel R...413, 1022 Chan, Lawrence...352, 358 Chancellor, Michael B Chanda, Diptiman...192, 206,

131 Abstract Author Index Chang, Alex H Chang, Bo Chang, Chia-Ming Chang, Guimin Chang, Heesoon Chang, Jin Woo Chang, Lung-Ji...775, 1045 Chang, Ming-Cheng...35 Chang, Patricia Chang, Seok Kee Chang, Shih-En Chang, Tammy Chang, Wen-Chung,...561, 581, 594, 663, 1051 Channon, Keith M...23 Chao, Hengjun...260, 263, 264 Chapdelaine, Pierre , 1081 Chaplin, Jennifer A Chapman, Robert L Chari, R Charrier, Sabine Chatani, Emi Chattopadhyay, Dalia Chavez, Christopher L...484, 957 Chavouin, Jean-Pierre Chekmasova, Alena A...245, 365, 571, 1043 Chen, Amy D Chen, Bing-Mae...36, 584 Chen, Chi-An...35 Chen, Chi-Yuan Chen, D.-T Chen, Dongquan Chen, Dung-Tsa Chen, Haifeng...82 Chen, Huang Chi Chen, Hunter H Chen, Irvin S. Y...224, 241, 540 Chen, Irwin...78 Chen, Jing Chen, Kuan-Hung Chen, Li...49, 963 Chen, Lingxia...3, 955 Chen, Liping...20, 253 Chen, May N Chen, Patrick Chen, Ping Chen, Steven C Chen, Weijun...25 Chen, Xuguang...508, 516 Chen, Y.-T Chen, Yi-Shen Chen, Yong Chen, Yu San Chen, Yuan-Tsong Chen, Yvonne Y Chen, Zhi-Ying...340, 341, 475 Cheng, Cheng Cheng, Jun Cheng, Seng H...31, 107, 342, 353, 676, 720, 747, 974, 987, , 1015, 1017, 1018 Cheng, Tao Cheng, Tian-Lu...36 Cheng, Weijun Cheng, Wen-Fang...35 Cheng, Yulan Cherel, Yan...742, 996 Cheung, Anthony T...346, 909 Chiang, Chi-Shiun Chiang, Yi-Shen Chiao, Paul L Chicoine, Louis G Chike-Obi, Chuma J Chikkanna-Gowda, C. P Chillon, Miguel...864, 881 Chin, Frederick T Chin, Joanna Y Chinnasamy, Dhana Chinnasamy, Nachimuthu Chino, Takenao Chiocca, Antonio E Chiocca, E. A Chiocca, E. Antonio Chiorini, Jay Chiorini, John A Chiu, Lu Tang

132 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Chiuchiolo, Maria Cho, Byoung Chul Cho, Eui-Sic Cho, Hong-Chan Cho, Kyoung-Sook Cho, Myung-Haing Cho, Namchul Cho, Sang Nae Cho, Sung-Ha Cho, Young-Hwa Choe, Han Choi, Bo-Ra Choi, Hye Choi, Hye-Jin Choi, Il-Kyu Choi, Jaemook Choi, Joon Sig...943, 946 Choi, Kyoung Baek Choi, Kyung-Ju Choi, Min Joung Choi, Soo-Min Choi, Uimook...322, 650 Chono, Hideto...314, 315, 575 Chorny, Michael Chouthai, Nitin Chow, Tak Kwong Vincent Chowdhury, Wasim H Chris, Mann Christensen, Camilla L Christensen, Jared E Christensen, Lane V...900, 1024 Christensen, Ulf B Christin, Anne Christine, Paul J Christov, Konstantin Chu, Datong...50 Chu, Qiuming...720, 747 Chuah, Marinee...300, 646 Chuang, Ching Kuang Chuck, Roy S Chung, Ching-Kuang Chung, Daniel...745, 819, 854 Chung, Hyesook...489, 492 Chung, Leland W. K Ciceri, F Ciceri, Fabio...37, 39 Cichutek, Klaus...218, 790, 813 Cingolani, Eugenio...60 Cipponi, Arcadi...37 Cirillo, Agostino Clapham, Paul R Clark, K. Reed...81, 136, 1075, 84, 823 Clark, Robert A Clarke, Christian...406, 1083, 1088 Clarke, Ian Clarke, Jennifer...31 Claudio, Maderna Clayman, G. L...45 Clemens, Paula R...662, 865 Cleveland, Matthew...8 Cleveland, Nicholas...8 Coady, Tristan H Coates, Allan L Cochrane, Melanie...103, 670 Cody, James J Coelho, Tiago H Coffin, Robert S Cohen, Justus B Cohen, Michael B...44 Cohen, Reuben Cohen, Richard N Cohen-Haguenauer, Odile Y...73 Cohn, Ellen F...260, 263, 264 Colella, Pasqualina...112, 746, 975 Coles, Rebecca L...420, 1018 Colle, Marie-Anne...742, 996 Collen, Désiré...99, 300, 333, 676 Colleoni, Silvia...321, 434, 1106 Colletti, Evan Collie, D. David S Collie, David S Collins, Margaret H Colloca, Stefano Colombi, S

133 Abstract Author Index Comijn, Elisabeth M Comijn, Lizzy...85, 88 Comin-Anduix, Begonya Comix-Anduix, Begonya Concia, Massimo Conese, Massimo Conley, Shannon M Conlon, Thomas...110, 983, 1054 Connault, Elisabeth Connelly, Jon P...58 Connole, Michelle Connor, Bronwen Constantinou, Varnavas Contreras, Juan L Cook, Todd G Cooney, Ronan Cooper, Laurence J , 372, 587 Cooper, Mark J...397, 518, 910 Copreni, Elena Corbella, Paola Cordelier, Pierre...204, 633, 1042, 1103 Corjon, Stephanie Cornel, Fraefal Cornetta, Kenneth...306, 308, 325, 969 Cornu, Tatjana I Cortese, Riccardo Cortez, Nenita Cossette, Travis Costanzi-Strauss, Eugenia Cotrim, Ana P...628, 671 Cotsarelis, George Cottin, Sylvine Cotugno, Gabriella...112, 975 Coulombe, Zoé Cousin, Béatrice Coutelle, Charles...23, 324, 344, 416 Couture, Larry...309, 312 Couture, Sylvana Cowan, Morton J Cox, Nancy R...727, 1002 Cox, Peter Cox, Timothy M Coyle, Christian H Cozzolino, Carmine Cradick, Thomas J Cress, Dean Crettaz, Julien...362, 831 Creutz, Heike Cripe, Timothy P...227, 394, 461, 462, 608 Crippa, F Cristillo, Anthony Crombleholme, Timothy M Cross, Scott Crowley, James Crowther, Carol...363, 707 Croyle, Maria...13, 42, 149 Cruz, Pedro E Cruz, Yolmari...18 Crystal, Ronald G...100, 158, 242, 293, 317, 356, 419, 582, 597,...655, 748, 752, 755, 979, 1109 Cuellar, Jason M Curcio, Claudia Curiel, David T...15, 33, 124, 437, 440, 621, 727, 862,...868, 872, 879, 880, 886, 890 Curran, Michael A Currier, Mark A...227, 462 Curry, William T Curtin, J Curtin, James F...34, 174, 176, 177, 181, 252 Czer, Peter Dai, Anlan Dai, ZhiYan Daly, Kieran Dames, Petra Dan, Kazuyuki Dandapat, Abhijit Daneshmand, Mani A D Angelo, Armando Daniel, Waterkamp...76 Daniels, Janice K Danko, Istvan Danos, Olivier...137, 881, 991 Darcy, Raphael Darlix, Jean-LUC

134 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Darmon, Christophe Darvish, Daniel Das Gupta, Tapas K David, Callejas David, Stefan Davidoff, Andrew M...103, 670 Davidson, Beverly L...57, 62, 695, 699, 724 Davie, Earl W Davies, Lee A...342, 420, 948, 1008, 1017, 1018, 1068 Davis, Adam...77 Davis, Corrine R Davis, Pamela B...508, 516, 517, 1067 Davydova, Julia Day, Terrence De, Bishnu P...655, 755 de Carvalho, Anna Carolina V de Cortie, Carin J De Geest, Bart...99, 333, 676 De Geest, Bruno G de Haan, H De Jesus, Michelle A de Jong, Albertine De Meyer, Simon F de Miguel, Fernando De Palma, Michele De Ravin, Suk See De Royer, Julien...73 de Silva, Suresh De Smedt, Stefaan C...490, 507, 902, 1020, 1069 De Waele, Liesbeth Dean, David A...474, 499, 501, 1006 Debelak, Daryn J...87 Debyser, Zeger Deckmyn, Hans Deglon, Nicole Deichmann, Annette Deisseroth, Albert...150, 163, 168 DeKelver, Russell C Dekker, Joe...13, 42 del Rio, Adolfo Del Vecchio, Luigi Delépine, Pascal...207, 905 Delker, Sebastian Della Valle, Patrizia Dell Osso, Lou Demaster, Amanda K Demeester, Jo Demeester, Joseph...507, 902, 1020, 1069 Deming, Timothy J DeMorest, Zachary Denby, Laura...619, 857 Denèfle, Patrice...412, 833 Denes, Bela Deng, Wentao Deppe, Herbert...21 Derakhshan, Faramarz Derecho, Ivy...309, 312 Derks, Sanne Dermody, Terence S Deroose, Christophe M Desai, Maya K Descamps, Delphyne Desmond, R. A...53 Desmond, R. M Desmond, Renee A Devaux, Patricia Dexi, Liu Deyle, David R Dhar, Debanjan Di Paolo, Nelson C Di Serio, Clelia...766, 1063 Di Stasi, A Di Vicino, Umberto...746, 999 Diaconu, Iulia Dias, Joao D...619, 632 Díaz-Nido, Javier...535, 1040 Dichek, David A Dickerson, David...66 Dickey, David D...83 Didier, Pete...27 Dietz, Allan B DiGiusto, David L Dijkstra, Marike H...46 Ding, Qingqing

135 Abstract Author Index Ding, Zhaobing Dirks, Peter DiRosario, Julianne Do, Yoonkyung Doan, Tuan Dobaño, Carlota Dobrenkov, Konstantin Doctor, Jessica H Doddy, Timothy G Dodge, James C...31, 987 Doering, Christopher B...407, 408 Doglioni, Claudio Dohmann, Hans F Dokter, Maarten Dolman, Carl Doloff, Joshua C Domb, Avi Domenici, Luciano Dominiczak, Anna F Donahue, Robert...241, 1091 Donate, Amy...18 Doneanu, Catalin Dong, Jian-Yun Dong, John...272, 457, 565 Dong, Ziming Donna, Stolz Donsante, Anthony...1 Doody, Timothy...502, 917, 919 Doolan, Denise L...16, 148, 153, 157 Dore, Franca Doremus, Caitlin...113, 409, 721 Dorey, Kathleen Doria, Monica Doronin, Konstantin Dorrie, Jan Dorset, Daniel C Dotti, G Dotti, Gianpietro...38 Doty, Raymond T Douar, Anne M Douar, Anne-Marie Dougherty, Christopher J Douglas, Joanne T Downey, Harre D Doxiadis, Ilias Draghia-Akli, Ruxandra...17, 146, 173, 754, 916 Draghici, Elena Dreicer, R...45 Dresch, Christiane Drescher, Charles...54, 200, 1111 Dresser, Betsy L Drew, Horace R Driessen, Wouter H Drocourt, D Drosatos, Konstantinos Du, Lily M Du, Ping Duan, Dongsheng...130, 133, 722 Duan, Donsheng Duchateau, Philippe Dudek, Arkadiusz Duffy, Lisa...306, 308 Dufour, Jason...27 Dufour, Noelle Duimel, Hans Dukart, Hillary Z Dunbar, Cynthia E...773, 1091 Dunphy, Nina T Dupouy, Sandra Dupré, Loïc...247, 648 Duran, Hebe A Durell, Karen L Dzojic, Helena Ebert, Oliver...49 Economou, James S Edamura, Kohei Edlund, Karin Edmonson, Shyrie...259, 839, 1085 Edwards, Carol C Egan, M. A Egawa, Shin Eggert, Brian C...93 Ehrhardt, Anja Ehsani, Ali

136 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Eichtinger, Magdalena Eigenbrod, Sabina Einfeld, David A Eklund, Aron Ekstrom, Tomas J...41 Elankumaran, Subbiah Eldridge, J. H Elfinger, Markus Elgadi, Mabrouk M Ellerhorst, Julie Ellinwood, N. Matthew...98, 996 Elliot, Shauna Ellis, James Elnitski, Laura Elojeimy, Saeed El-Sayed, Ayman Ely, Abdullah Embury, Jennifer E Emery, David W...70, 692, 778, 1062 Encinas, Tony Endo, Hitomi Endo, Masayuki...131, 256, 290, 295, 410, 691 Endo, Yoko Engbersen, Johan F. J Engel, Barbera Engelhardt, John F...385, 719, 848 Engler, Tatjana...10, 336 Epperly, Michael W...199, 203, 302, 475 Erbacher, Patrick Erickson, David C Eriksen, Jens...500, 914 Eriksson, Minna...216, 623, 632 Erlichman, Charles...47 Escourrou, Jean Escriou, Virginie Esfandyari, Tuba...706, 1026 Esposito, Nicola Essand, Magnus Estmer Nilsson, Camilla Estrada, Marvin Ettyreddy, Damodar...148, 753, 884, 891 Evelegh, Carole Everhart, Drew Everts, Maaike...124, 872 Excoffon, Katherine J. D. A...83, 335, 809 Eymard, Bruno Fabre, Emmanuelle Facchini, Giulia Faella, Armida Fairbanks, Carolyn Fakhouri, Tamer...174, 176 Fall, Abbie A...63 Fall, Brent Fallaux, Frits J Fallon, John T...49 Fan, Jun Y Fan, Sheung Tat Fang, Juan Farassati, Faris...706, 1026 Farley, Raymond Farrar, G. Jane Farrell, Christopher J Farzaneh, Farivar Farzaneh, Farzin Fassas, Athanasios Fatima, Bosch Fattal, Elias Faubion, Mindi H Faulkner, Louisa M Faull, Richard L. M Favaro, Patricia Fawcett, Paul Fechner, Henry...694, 847, 851 Federici, Thais...77, 284, 531, 677 Federico, Mingozzi Federoff, Howard J...30, 435, 458, 539 Fehlings, Michael G Feijen, J Feijen, Jan Felber, Barbara Feldman, Eva L...270, 677 Feldmann, Heinz Felix, Garcia Felller, Stephanie

137 Abstract Author Index Fellows, Wendy...55 Felsburg, Peter J Felsenfeld, Gary Felsher, Dean Felton, Kathleen E. A Feng, Yingmei...99, 333, 676 Fenjves, Elizabeth S Férec, Claude...207, 905 Feril, Jr., Loreto B Ferkol, Thomas W Fernandes, Julio C...553, 904 Fernandez, Ester Fernández-Ruiz, Verónica Fernando, Abarzua Ferrari, Giuliana...250, 651, 1060 Ferrari, M Ferraro, Bernadette...18 Ferreira, L. M Ferrer, Mireia...864, 881 Fetoni, Annarita Fewell, Jason G...170, 182 Fichter, Katye M Fidler, Jonathan A Figueiredo, Marxa L Figueredo, Joanita...155, 255, 257, 366, 574 Filareto, A...64 Fina, D...64 Finegold, Milton...5, 417, 522, 840, 964 Finka, Andrija Finn, Eric...113, 127, 409, 721, 1022, 1076, 1078 Finn, Jonathan D Fischer, Anne C Fischer, Anthony J Fishbein, Ilia Fisher, Jeanne...27 Fisher, Kerry Fiszman, Marc Y Flaat, Mette...27 Flake, Alan W...131, 256, 290, 295, 297, 410, 691 Flannery, John G Fleckenstein, Katharina Fleeton, Marina N Fleischmann, Bernd K Fleshner, Neil...48 Fletcher, James...66, 800 Fletcher, Susan...63 Fliesler, Steve J Flindt, Esben N Flotte, Terence...110, 718, 820, 990, 1045, 1054 Fodor, Istvan Fodor, Nadja Foley, Joe W Foley, Joseph W Follenzi, Antonia Fong, Y Fontana, Raffaella C...37 Fontanellas, Antonio Forde, Patrick F...917, 920 Forero, Adriana...55 Forgez, Patricia Forman, Stephen J Formisano, Pietro Forni, Guido Forsayeth, John Fortes, Puri Foster, A Foster, Aaron...38 Foster, Edmund Fougerousse, Francoise Fouquet, Francoise Foust, Kevin D Fowler, Daniel H...48 Franciola, Darcy...199, 475 Francis, Jennelle Frankel, Gad Fratantoni, Joseph C...185, 431 Fratini, Paula Frazer, Maria E Frederik, Peter Freese, A. B Frenzke, Marie E Freson, Kathleen Friedmann, Theodore...143, 1048 Friesicke, Matthias

138 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Frndova, Helena Froehner, Stan Froehner, Stanley C Fromes, Yves Fruehauf, Stefan...603, 634, 649, 828, 1059 Fu, Haiyan...97, 824 Fuchigami, Tetsuya Fuchita, Michi Fueglistaler, Philipp Fuess, Sally...4 Fuji-Funeshima, Naoko...450, 684 Fujii, Masazumi Fujikawa, Satoshi...795, 1010 Fujiki, Minoru Fujimoto, Kiyohide Fujimoto, Koji Fujimuro, Masahiro Fujisawa, Masato...631, 729 Fujiu, Katsuhito Fujiwara, Toshiya Fujiwara, Toshiyoshi...384, 446, 600, 612, 763 Fukuda, Shigekazu Fukunaga, Yoshitaka Fukuyama, Ryuichi Fulgham, Pat Fuller, Jessica...105, 106 Fuller, Stephen J Fultz, Megan...860, 891 Fuminori, Teraishi Fung, Kevin Funke, Sabrina Furlani, Dario Futaki, Shiroh Gabrovsky, Vanessa...337, 484, 542, 957 Gaddy, Daniel F Galan, Jorge E Galanis, Evanthia...47, 51 Gall, Jason...884, 891 Galla, Melanie...327, 805 Gallagher, Patrick G Gallego-Hernández, M. Teresa...535, 1040 Galli, Cesare...321, 434, 1106 Galli, Rossella Gallo Stampino, C Galula, Jennifer Galy, Anne...247, 648, 803 Gambhir, Sanjiv S...237, 596, 1051 Gambotto, Andra Gambotto, Andrea...546, 564 Gan, Ren-bao Ganesh, Shanthi Gangadharan, Bagirath...407, 408 Gangeswaran, Ruthi Gansemer, Nicholas D...335, 809 Gao, Gaung-Ping Gao, Guang-Ping...8, 574, 661 Gao, Jun Gao, Mei Hua Gao, Xiang Gao, Yi Gao, Yongxin...275, 360, 1025 Garabedian, Elizabeth K García, Ana...535, 1040 Garcia, Luis Garcia Hoyos, Maria Garcia-Bañuelos, Jesus Garcia-Blanco, Mariano A García-Escudero, Vega...208, 535, 1040 García-Sastre, Adolfo...49 Gardner, Thomas A...56, 626, 729 Gargini, Ricardo...208, 1040 Gargiulo, Annagiusi Garlington, Wendy Garvik, Barbara Gasmi, Mehdi Gauldie, Jack Gaussin, Armelle...73 Ge, Rongbin Ge, Ying Geddes, Duncan M Gee, Adrian P Geel, Marieke Gehl, Julie...19, 500, 679, 914 Gehlbach, Peter L

139 Abstract Author Index Gelovani, Juri G...361, 395 Genovese, Pietro Gentner, Bernhard...434, 774 Gentsch, Marcus Georgomanoli, Maria Gerard, Christopher J...87 Gerard, Daly Germain, Sean Gerritsen, Winald R Gerson, Stanton L...15 Gerull, Sabine Gess, Austen Getty, Robert Geurts, Aron M Ghaffari, Seyed Hamidolah Ghani, Karim Gharwan, Helen Ghavamzadeh, Ardeshir...291, 1047 Ghodis, Pantea Ghosh, Arka Ghosh, Arkasubhra Ghosn, Bilal...175, 932 Giacca, Mauro...6, 120, 357, 980 Giacco, Ferdinando Giachelli, Cecilia M Giandomenico, Valeria Giangrande, Paloma H Giannetti, Andre Gianni-Barrera, Roberto Giannoukakis, Nick , 1105 Gibbons, Douglas Giddings, Angela M Giering, Jeffery C...59 Gil, Estel...864, 881 Gilbert, Merle L Gilbert, Richard...18 Gilham, David E...371, 599 Gill, Deborah G Gill, Deborah R...342, 420, 948, 1008, 1015, 1017, 1068 Gillespie, Rebecca A...227, 462 Gillijns, Veerle Gillis, Kimberly Gilmore, Brian L Giordano, Frank A Giordano Attianese, Greta M. P...38 Giorgio, Todd D Gissel, Hanne...19, 679 Gjata, Bernard Gjetting, Torben...483, 494 Glasgow, Joel N...124, 862, 890 Glazer, Peter M...276, 703, 708, 1099 Gleich, Bernhard Glenn, Andrew Glimm, Hanno Gloria, Gonzalez-Aseguinolaza Glorioso, Joseph...28, 32, 55, 215, 310, 986, 1000 Glover, Dominic J Goda, Kazumasa Goetzmann, Eric Gofrit, Ofer Goins, William F...215, 310, 986 Gojkovic, Zoran...41 Gold, Richard Golde, William T Goldsmith, Corinne M Goldsmith, Corrine M Golzio, Muriel Gomes, Erica M Gomes, Heitor C Gomez, Martha C Gomez, Raquel Gonzalez, Jaime Gonzalez, Melissa Gonzalez-Aseguinolaza, Gloria...362, 469 Gonzalez-Cuevas, Jaime J Gooch, Christina...66, 323 Goodwin, W. J...45 Goonetilleke, Nilu Gopalan, Began...197, 1097 Gorbatyuk, Oleg...25 Gordon, Catherine Gori, Jennifer L...235, 656, 1044 Goss, James...28, 32, 1000 Gotoh, Akinobu...438, 631,

140 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Gould, Peter V Gouras, Peter Govindasamy, Lakshmanan Grabherr, Reinhard Graf, Marcus Graham, Frank L Grandi, Paola...55 Grant, Maria Grant, Rebecca...661, 743, 1086 Grassman, Elke...246, 805, 1061 Gratton, Michael A Gray, John Gray, Michael...149, 757 Green, Albert Green, Anne-Marie , 1018 Greenberg, Kenneth P Greenberg, Philip D Greenberger, Joel S...199, 203, 302, 475 Greenberger, Shoshana Gregorevic, Paul...127, 409, 721, 1076, 1078 Gregory, Carl Gregory, Philip D...32, 321, 373, 426, 436, 585, 776, 777 Greiser, Udo Grenman, Reidar...632, 635 Grez, Manuel Grier, Richard Griesenbach, Uta , 1017 Griffin, Diane E Griffith, Thomas S...44, 595 Griffiths, Denise A...31 Grimm, Dirk...59, 693, 977, 1104 Grimm, Elizabeth Grinstaff, Mark W...906, 908 Grist, Thomas M Gritti, Angela...96, 423, 434, 1106 Grizot, Sylvestre Grompe, Markus...5, 522, 840 Gross, Fabian Gross, Jennifer Grossheim, Jonathan Grossman, William Groth, Amy C...692, 778 Grubman, Marvin J Gruszczynski, Carole Gu, Yuchen Gu, Yu-chen Guan, Yi Guerciolini, Roberto Guertin, L...45 Guggino, William B...719, 820 Guglielmi, Kristen M Guhl, Eva Guidotti, Luca G Gulick, James Gumlaw, Nathan Gunning, Chris Guntaka, Ramareddy V Gunther, Roland Guo, Chunlu Guo, Tracy Gupta, Sanjeev Gurke, Lorenz Guschin, Dmitry Guse, K Guse, Kilian...610, 216, 619, 623, 632 Gustafson, Elisabet Haanen, John Haase, Georg...29 Haast, Saskia...85, 88 Haba, Ryota...450, 684 Habib, Nagy...629, 837 Habu, Yuichiro Hacker, Caroline V...71 Hackett, Christopher S Hackett, Neil R...100, 317, 356, 582, 597, 655, 748, 755, 979 Hackett, Perry B...24, 109, 220, 339, 481, 482, 912, 1070 Haczek, Cornelia...21 Hadac, Elizabeth...52, 764 Hadaczek, Piotr...435, 724 Haddad, Hafedh Haga, Kazunori...438, 762 Hagstrom, James E Hai, Mehreen...243, 771 Haidet, Amanda M

141 Abstract Author Index Haisma, Haisma J Haisma, Hidde J...46, 230 Hajitou, Amin Hakkarainen, T...53, 219 Hakkarainen, Tanja...216, 601, 617, 623, 639 Hakobyan, Narine Hakumäki, Juhana M Halbert, Christine L...418, 1009 Hales, Laura M Hallek, Michael...75 Hallenbeck, Paul L Hama, Susumu...400, 897 Hamada, Hirofumi Hamada, Katsuyuki...631, 731 Hamilton, James P Hamm, J...45 Hammel, Pascal Hammer, Robert P Hammond, H. Kirk Hampton, Thomas G Han, Jian Han, Jiang Han, Sang W...477, 488, 554 Han, Tie...878, 886 Han, Zongchao...829, 846, 855 Hanawa, Hideki Hanchett, Kathy Handlos, Vagn N Handy, Chalonda...31, 1077 Hanks, Brent A Hanna-Riikka, Karkkainen A Hannay, Jonathan Hannun, Yusuf Hara, Hidehiko Hara, Isao Hara, Kazuhiro Harada, Akiko Harada, Taishi Harada, Yui Haraguchi, Miki...129, 134, 721, 1078 Harashima, Hideyoshi...498, 704, 893, 896, 897, 899, 901, 949 Harats, Dror Harbottle, Richard P...23, 344 Hardcastle, Jayson Harden, Paul Harding, Penny M...63 Harding, Sian E Hargrove, Hargrove W Hargrove, Phillip Harkins, Richard...221, 285 Harmeling, Benjamin R Harnaha, Jo Harold, Nancy Harper, Scott Q...57, 695, 699 Harriosn, Phillip Harris, Chad E Harris, Jennifer Harrison, Rachael Harsch, Kelley Harshima, Hideyoshi Hart, Stephen L Hartl, Dominic Hartman, Zachary Harui, Airi Harvey, Ben-Gary Harvey, Brandon K Hasbrouck, Nicole Hasegawa, Kouichi Hasegawa, Mamoru...184, 190, 381, 787, 795, 1010, 1017 Hashimoto, Kouichi Hashimoto, Yuuri...384, 600, 612, 763 Hasholt, Lis Haskins, Mark...26, 72, 104, 351, 650 Hatakeyama, Hiroto Hatashima, Hideyoshi Hattori, Yaka Hauck, Bern...90, 91 Hauck, Bernd...3, 432 Hauck, Berndt Hauswirth, William W...433, 983 Hautaniemi, S Havenga, Menzo J Hawes, Norman L Hawkins, Peter

142 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Hawkins, Robert E...371, 599 Hawley, Robert G Hawley, Teresa S Hayakawa, Takao Hayden, Michael R...88 He, Cheng-Yi He, Chen-Yi He, Ming-liang He, Suiwen He, Weijing He, Xianghui...487, 1033 Heald, Alison E Heard, Jean-Michel...29, 996 Heavener, S. Brett Hebbel, Robert P Heberer, Michael Hebert, Michael Hecker, James G...398, 514 Hefferan, Michael P Hegan, Denise Hege, Kristen Hegge, Julia O Heikura, Tommi Heinonen, Seppo Heiss, Christian Helfrich, Wijnand...46 Heller, Loree C...18 Heller, Richard...18 Hematti, Peiman Hemminki, A...53, 219 Hemminki, Akseki Hemminki, Akseli...216, 601, 610, 617, 623, 632, 639 Henkelman, Mark Henning, Petra Henry, Rebecca A Henthorn, Paula S...72 Heo, Hwon Herlihy, John D Hermens, Wim...85, 86, 88, 348 Hermiston, Terry...221, 285 Hermonat, Paul L...40, 115, 316, 827, 832, 852 Hernandez, Reuben Hernandez Prata, Carla A...906, 908 Herrero, Ignacio Hershfield, Michael S Herskind, Carsten...634, 828 Herson, Serge Heslop, Helen E...38, 368, 375 Hesse, Andrea...441, 885 Hester, Mark...31 Heston, Warren Hiatt, Peter Hibbitt, Olivia C...23, 391 Hickey, Melissa A Hickman, Miranda Hickstein, Dennis D...243, 771 Hidaka, Chisa Hideyuki, Yamashita Higashimoto, Tomoyasu Higginbotham, Mary L High, Katherine...7, 90, 91, 259, 432, 525, 745, 839,...843, 956, 1056, 1085, 1089 Hilligoss, Dianne Hinata, Nobuyuki Hinderlich, Stephan Hines, Ian N Hinkel, Rabea Hirai, Yukihiko Hirano, Hisao Hirao, Lauren...146, 751, 754 Hirao, Yoshihiko Hiraoka, Kei Hirata, Aiko Hirata, Fusao Hirata, Roli K...422, 1037 Hiratsuka, Masaharu Hiriyanna, Suja Hironaka, Takashi Hirose, Kiyomi...493, 710 Hiroshi, Fukuhara Hirsch, Matt Hirsch, Matthew L Hisanaga, Michiyoshi Hisano, Terumasa

143 Abstract Author Index Hitt, Mary M Ho, Ivy A. W...463, 620 Ho, Seong-Hyun Ho, Yi-Ping Hoare, Melissa Hochberg, Abraham Hocquemiller, Michäel...29 Hodge, Gregory Hodges, Bradley Hodgson, Clague Hoel, Brian D...272, 457, 565 Hoelzer, Dieter Hoffmann, Florian Hofmann, Andreas...696, 804 HogenEsch, Harm Hogikyan, Norman D Hogrel, Jean-Yves Hokey, David A...280, 751 Hollyman, Daniel Holm, Per S Holman, David H Holmes, Michael C...321, 373, 426, 436, 777 Holzmueller, Regina Hong, Bum Kee Hong, Saw See Hong, Youngtae...74 Honig, Sarah B...34, 181 Hopkins, Tim Horinaga, Minoru Hosey, James Hoshi, Minako Hoshiya, Hidetoshi Hosseinkhani, Hossein Hossienkhani, Mohsen Hou, Donna Y Howard, Douglas B Howard, Linda Howe, Steve Howe, Steven J...324, 416, 1013, 1058 Hsiao, Pen-Wen Hsieh, Chang-Yao...35 Hsu, Alice...686, 912 Hsu, Chi...439, 884 Hsu, David...309, 312 Hsu, Eric C...346, 909 Hsu, Hui-Chen Hsu, Ya-Hsuan Hu, Changping Hu, Fang...50 Hu, Guohong Hu, Jim Hu, Shiu-Lok Hu, Xiaoxia Hu, Yu-Chen...466, 471, 550 Hu, Zhongbo Huang, Jing-Hua Huang, Joanne Huang, Leaf Huang, Li-Jiau Huang, Louis Huang, Shaohua , 32, 986 Huang, Shaoling Huang, Wen-Ching Huang, Yu Huang, Yu-Hung...43 Hubbell, Jeffrey A Huber, Anke...75 Huber, Hans Hubner, Ralf H...979, 1109 Hubner, Ralf-Harto Hughes, Christina F Hughes, Stephanie M Huh, Nam-ho Huhtala, Tuulia Hui, Daniel...259, 1056, 1085, 1089 Hui, Kam M...463, 620 Huizenga, Aafke Humeau, Laurent Humme, Sibille...75 Humphries, Margaret Humphries, Peter Humphries, R. Keith Hung, Mien-Chie Huo, Longfei

144 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Huo, Yunwen Huss, Thierry Huusko, Jenni M Huyn, Steven T Huynh, H Hwang, Sally...65, 551 Hwang, Shiaw-Min Hwang, Soon Kyung Hyde, Stephen C...342, 420, 948, 1008, 1015, 1017, 1018, 1068 Hynes, Sean O Hyun, Jong-Pil Iacomini, John , 1091 Iankov, Ianko...47, 51 Ibañez, Irene L Icyuz, Mert Idamakanti, Neeraja Ide, Lucienne M...407, 408 Ieronimakis, Nicholas...139, 970, 1110 Igarashi, Katsuhide Ihalainen, Teemu I Iida, Akihiro Iida, Takayuki Ijiro, Kuniharu Ijuin, Moeko Ikeda, Yasuhiro Ilaria, Visigalli Imam, Syed Z Inagaki, Katsuya...4, 387 Ingle, James N...47 Ino, Yasushi Inoue, Hideo Inoue, Hiroyuki Inoue, Makoto...184, 190, 381, 787, 795, 1010, 1017 Inouye, Masayori Iodice, Carolina Iqball, Sharifah Ireland, Joanna Irvin, Tracie...743, 1086 Irvine, Scott A Irwin, Richard D Isayeva, Tatyana...206, 760 Ishida, Akihiko Ishida, Kumi Ishida, Tatsuhiro...231, 493, 710 Ishii, Keisuke Ishii, Ken Ishiwata, Akira Ishizaki, Masamichi Ishizaki, Masatoshi Israel, Yedy Issa, Mohamed M Itaka, Keiji Ito, Akihito Ito, Kaori Ito, Mayumi Ito, Takayuki...359, 761 Ito, Tetsuo Ito, Tomoko...936, 937 Iversen, Patrick...63 Ivics, Zoltan Iwasaki, Hitoshi...795, 1010 Izquierdo, Marta Izzo, Barbara Jackson, Mark W Jacobs, Frank...99, 333, 676 Jacobs, Jonathan Jacobs, Mike A...66 Jacobs, William R Jacqmarcq, Cécile Jaffredo, Thierry Jakobsen, Maria...69, 793, 1028 James, Scott E Jamieson, Andrew C Jamieson, Leanne Janat-Amsbury, Margit M Janelsins, Michelle C Jang, Jiwon...74 Jang, Kyoung Jin Janik, John E Jans, David A...510, 740, 925 Janssen, Paul M Jarjour, Jordan Jaroszeski, Mark J...18 Jaruga, Ewa

145 Abstract Author Index Jason, Gall G. D Jasti, Aparna Jay, Chris...141, 222, 687, 1021, 1102 Jeanneau, Laetitia Jeanson-Leh, Laurence Jeng, Meei-Huey...56 Jennifer, McDonnell Jennings, Jerillyn S Jens, Jackie K Jensen, Bo S...41 Jensen, Eric S Jensen, Michael C...370, 372, 373, 561, 581, 594, 596,...598, 663, 780, 1051 Jensen, Ryan...81, 1075 Jensen, Thomas G...483, 494, 712 Jeon, Eugene Jeon, Hyun Jeong Jeong, Jae-Gyun Jeong, Ji Hoon Jeong, Jin Sook Jeong, Moonsup Jeong, Sunjoo...643, 835 Jerabek-Willemsen, Moran Ji, Won-Jin Jia, Zhen Jian, Weiguo Jiang, Canwen...353, 974 Jiang, Gang Jiang, Haiyan Jiang, Nan...275, 1025 Jiang, Xuan Jimenez, Gretchen...145, 756 Jimenez, Juan A Jin, Fang Jin, Xu Jin, Zhe Jo, Eui-Cheol Joel, Montane Joh, Ju Johannes, Czernin John, Gray T Johnson, Arthur T Johnson, Jarrod S...386, 389 Johnson, Kyle Johnson, Mai...233, 627 Johnson, Philip R...81, 84, 151, 159 Johnson, R. Paul Johnston, Grace Johnston, Julie C Johnstone, Brian H Jones, Brian H Jones, Frederick S Jones, W. Keith Jonghwan, Kim Jooss, Karin...602, 726 Jorritsma, Annelies Joseph, Guiandre Josien, Regis...34 Joudi, Fadi N...44 Jounaidi, Youssef Joung, J. Keith...58, 781 Jouvenot, Yann...32 Joyce, Sonali Juanarena, Nerea Judge, Luke M...113, 126, 409 Juergen, Kleinschmidt A...76 Juliusson, Bengt...41 Jullienne, Betsy Jun, Byungdug June, Carl H Jung, Cindy Jung, Juyeon Jung, Sung-Mi Jung, W. Edward Junger, Kerstin Junghans, Richard P...266, 577 Jure-Kunkel, Maria N Kaar, Joel Kaddur, Kadija Kadmon, Dov Kafri, Tal...782, 838 Kagawa, Shunsuke...384, 600, 612, 763 Kagiya, Go Kai, Yoshiteru

146 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Kaikkonen, Minna...393, 464, 467 Kaiser, Julia Kaiser, Larry Kajitani, Naoyo Kajiwara, Hidetoshi...353, 974 Kakimoto, Shinji Kakinohana, Osamu Kale, Ajit Kaliberov, Sergey A Kaliberova, Lyudmila N Kalle, Christof von Kalos, Michael Kaloyannidis, Panayotis Kaltoft, Keld Kalyuzhniy, Oleksandr...863, 871 Kamata, Masakazu...241, 540 Kambara, Hiro Kamen, Amine...89, 430 Kamida, Tohru Kamidono, Sadao Kamiya, Hiroyuki...498, 949 Kamizono, Junichi Kampgen, Eckhart Kamps, Gera K Kamps, Jan A. A. M Kan, On Kan, Takatsugu Kan, Yuet W Kanaoka, Masaharu...14 Kandan, Aravindaram Kaneda, Yasufumi...971, 1112 Kanegae, Yumi...14, 281 Kanehiro, Hiromichi Kaneko, S Kaneko, Shin...39 Kanerva, A...53, 219 Kanerva, Anna...216, 601, 610, 617, 619, 623 Kang, Bong-Su Kang, Elizabeth M Kang, Hyoung Jin...74 Kang, Jeong-Hun Kang, Lu...93, 97, 824 Kang, Seok Kang, Yoona Kanno, Jun Kantor, Boris Kao, Chinghai...56, 626, 729 Kapoor, Veena Karahanian, Eduardo Kari, Airenne J Kariath, Anu Karkkainen, Hanna-Riikka A Karlen, Andrea...235, 656 Karlsson, Stefan Karponi, Garyfalia Karrasch, M Kasahara, Noriyuki...438, 762, 796 Kasai, Kazue Kaslow, David C...145, 756 Kaspar, Brian K...31, 1077 Kastelein, John J...348, 977 Kasturi, Sudhir P Katakam, Anand K Kataoka, Kazunori Kataoka, Miho...299, 958 Katav, Avi Katayama, Yoshiki...511, 520, 933, 1071 Katherin, High A Kato, Ikunoshin...315, 575 Kato, Ikunosin Kato, Ryuichi Kato, Tomonori Katsoupi, Polyxeni Kaufman, Christopher D Kaufman, Randal J Kaul, Rajinder...66 Kaur, Balveen Kawa, Milosz P Kawabata, Kenji...669, 859, 861, 875, 882 Kawai, Toshinao Kawamura, Hitoshi...443, 446, 600, 728 Kawamura, Kiyoko Kawano, Ryoko Kawano, Takahito...520,

147 Abstract Author Index Kay, Mark...4, 5, 59, 338,...340, 341, 387, 475, 665, 693, 840, 977, 1104 Kaye, David M Kazazian, Jr, Haig H Kazuki, Yasuhiro Keane, Thomas Keating, Kenneth Keck, Kathy Kehlenbeck, Sylvia Keiser, Nicholas Keller, Daniela Kells, Adrian P Kelly, Elizabeth...52, 764 Kelly, Meagan E...260, 263, 264 Kenna, Paul F Kennedy, Douglas R...650, 769 Kepes, Steven Keramaris, Elizabeth Keravala, Annahita...337, 484, 957 Keuling, Angela M Keyser, Kirsten Khademhosseini, Ali Khai, Ngin Cin Khalil, Ikramy A...893, 894, 897 Khalili, Mitra Khan, Amir S...17, 146, 173, 754, 916 Khan, Junaid Khan, Zahidul...41 Kharazi, Pejman Kharaziha, Pedram Khare, Pranay D Khazi, Fayaz...2 Khoury, Maroun Khu, Naw H Khurana, Tejvir S Kiang, Anne...9, 11 Kieffer, Timothy J Kiem, Hans-Peter...66, 239, 323, 378, 424, 562, 772, 778, 799, 800 Kikuchi, Hiroshi...493, 896 Kikuchi, Satoru Kikuchi, Yasushi Kim, Byong-Moon Kim, Chae-Young Kim, Dokeun Kim, Dong-Eun Kim, Eun S Kim, Hee-Jong Kim, Hyun Ah...268, 273 Kim, Hyunsoo Kim, In-Hoo Kim, In-Wook Kim, Jaehyung Kim, Jae-Sung Kim, Ji Yun Kim, Jin Wook Kim, JinSun Kim, Ji-seon Kim, JiYun Kim, Jongheon Kim, Jong-Man Kim, Jong-Mook Kim, Joohang Kim, Joo-Hang...223, 228, 593, 615, 638 Kim, Joong Gon...74 Kim, Jumi Kim, Jung Seok Kim, Kenneth...426, 436 Kim, Keun-Sik Kim, Kevin Kim, Kinam Kim, Ki-Sun Kim, Kyunghwa...273, 943 Kim, Mee Young Kim, Min Jung Kim, Min-Ju Kim, Oak Hee Kim, Pyung-Hwan Kim, Samuel Kim, Seung Taik Kim, Sujeong...74, 575 Kim, Su-Jeong Kim, Sun Hee Kim, Sung Jin...544, 835, 853, 1036 Kim, Sung Wan...268, 273, 900,

148 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Kim, Sunyoung...74, 575 Kim, Tae Gyun Kim, Won Jong Kim, Wun-Jae Kim, Yeun-Ju Kim, Yoon Sang Kim, Yoon-Jin Kim Kwon, Yunhee Kimata, Yoshihiro Kimmelman, Jonathan...537, 968 Kimura, En Kimura, Hiromitsu...450, 684 Kimura, Shigemi Kimura, Takahiro Kimura, Toru...14 King, C. R...16, 148, 153 King, C. Richter...860, 891 King, G. D King, Gwendalyn D...34, 176, 181 King, Rick Kingsman, Susan M...71 Kinnon, Christine Kinoh, Hiroaki...190, 381 Kirkegaard, Erin S Kirschner, Richard E Kitajima, Masayuki Kiuru, Maija Kiviat, Nancy...54 Kiwada, Hiroshi...231, 493, 710 Kizana, Eddy...60 Kizuki, Shinji Kjeken, R Kjeken, Rune...161, 503, 504 Klatzmann, David Klausner, Eytan A Klein, Jason J Kleinschmidt, Juergen A...649, 978 Kline, Karen L Klinkenberg, David...55 Kloke, Björn-Philipp Klopsch, Christian Kluxen, Felix M Knippertz, Ilka Knoblauch, Christine Knoess, Sabine...770, 1061 Knop, David...92 Knutson, Russ Ko, Derek Ko, Shuwen Ko, Yong Kobasa, Darwyn...155, 162, 757 Kobayashi, Akihiko Kobayashi, Hidenori Kobayashi, Hideo...493, 896 Kobinger, Gary P...149, 155, 162, 757 Kochanek, Stefan...10, 336, 865 Kochanek, Stephan Kodaki, Tsutomu Kodera, Mika Koeberl, Dwight D...107, 349, 352, 414 Koerber, James T...80 Kogure, Kentaro...893, 894, 896, 897, 899, 901 Koh, Shan Koh, Shuwen Koh, Yoewook Kohn, Donald B...249, 775 Koike, Hiromi Koizumi, Naoya Kojanyan, Marine Kojima, Toru...384, 763 Koldej, Rachel...68 Kolk, Andreas...21 Komai, Michael Komaromy, Andras M Komarova, Svetlana...15, 879 Komaru, Atsushi Komatsu, Norio Komatsubara, Kim Komiya, Kazuo...14 Komiya, Setsuro Kon, Takashi Kondo, Haruhiko Kondo, Saki Kondo, Takashi

149 Abstract Author Index Kondrikova, Galina...25 Konety, Badrinath R...44 Kong, Hun-Young Kong, Jian Kong, Yanhong Koniar, Brenda Konovalova, Svetlana...148, 153 Koo, Kyung Hye Koop, Sue...306, 308 Koota, Suvi E Koppanati, Bhanu Munil Kornegay, Joe N...130, 722 Kosai, Ken-ichiro Koster, Marla Kowalczyk, Tomasz H Koya, Richard C Koyama, Yoshikazu...401, 552 Koyama, Yoshiyuki...936, 937 Kozink, Daniel M Kozlov, Mikhail Kramer, Gabriela Kramer, Richard H Krance, Robert A Krasnykh, Victor Kratzke, Robert Krause, Anja...242, 1109 Kren, Betsy T Krendelchtchikova, Valentina Kreppel, Florian...10, 336, 865 Kretschmer, Peter J Kretzschmar, Hans Krieger, Thorsten Krishnamurty, Akshay Krisky, David...28, 32, 55, 310, 1000 Kristiansen, Karsten Kroeger, K. M Kroeger, Kurt M...34, 176, 177, 252, 538, 758, 887 Krogsdam, Anne Krouse, Allen E Kroutov, Vadim Krueger, Karen Kruse, Meghan A Krutilina, Nina Kuan, Jean...276, 703 Kubo, Chikage Kubo, Shuji...438, 631 Kudo, Asako...894, 899 Kudrolli, Tarana A Kuehn, Michael S...70 Kuhn, Irene Kuhn, Joseph Kuhr, Christian S Kuivenhoven, Jan Albert...348, 977 Kullmer, Martin...21 Kulomaa, Markku S Kumar, Padmasini...443, 614, 687, 1021, 1102 Kumar, Prasanna Kumar, Sanjay...192, 206, 549 Kumar-Singh, Rajendra...709, 995 Kume, Akihiro...86, 108, 359, 761, 836 Kumon, Hiromi Kung, Hsiang-fu...234, 609 Kuo, Chi-Chung Kuo, Hsu-Ping Kuo, Sheng-Chu Kuo, Tsun Yung Kupatt, Christian Kuppuswamy, Mohan N Kurachi, Shinnosuke...875, 882 Kurihara, Ryohsuke Kurimasa, Akihiro Kurinami, Hitomi Kurita, Ryo...657, 787 Kuroda, Shinji...384, 612, 763 Kuroiwa, Toshihiko Kuroki, Masahide Kurozumi, Kazuhiko Kurre, Peter Kurreck, Jens...694, 851 Kushwah, Rahul Kushwaha, Rampratap P Kusuda, Jun Kutner, Robert H...425, 531, 816 Kutzler, Michele A...147,

150 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Kuzniar, Tomasz J Kvam, Erik Kwon, Heechung Kwon, Minjeong...489, 492 Kwon, O.-Jun Kwon, Sang...906, 908 Kwon, Yong-Sam Kypreos, Kyriakos E La Perle, Krista La Seta, Simona...39 LaBresh, Kurt Lackner, Andrew...27 Laddy, Dominick J Laderman, Ken A Laderman, Kenneth A Laflamme, Karina Laforet, Pascal Lagenaur, Carl Lai, Meizan Lai, Ming-Derg Laing, Jennifer M...459, 472 Laipis, Philp J Laitinen, Olli H Laje, Pablo Lakomy, Robert Lali, Medina-Kauwe Lalor, Peggy Lam, Paula Y. P...463, 620 Lam, Siu-ling Lamont, Jeffrey Lan, Chun Wen...156, 164 Lan, Michael S Lan, Zhang Landre, Julien Lang, Jing-Yu Lang, Xiaoou Lange, Amy M Langer, Robert...43 Langridge, William H. R Langtry, Sarah Lantz, Larry M Lappalainen, Jari P Lapteva, Natalia Larchian, William Laroche, Serge Larocque, Daniel...34, 176, 252 Larson, Steve Larsson, Anita Lash, Lawrence Lattanzi, Wanda Lauer, Ulrich M Laufs, Stephanie...603, 634, 1059 Laura, Tononi Laurema, Anniina Lavilla-Alonso, Sergio Law, Mark...358, 964 Lawrence, Theodore S Lawson, Greg W Lawton, Anna E...342, 1008, 1018 Le, Long P...880, 886 Le Doux, Joseph M Le Gall, Tony...207, 905 Le Goupil, Delphine Leapley, Alyssa C Lebherz, Corinna Leduc, Sophie Lee, Ai Ping Lee, Benjamin H Lee, Boyoung Lee, Brendan...406, 1083, 1088 Lee, C. Chang I...298, 556 Lee, Chang G Lee, Che-Hsin Lee, Chen-Hsien Lee, Chien-Te Lee, Gary...373, 426, 436, 442, 585 Lee, Han Saem Lee, Han-Na Lee, Heuiran...544, 643, 835, 853, 1036 Lee, Hisoon Lee, Hong-Kyu Lee, Hsiao-Ping Lee, Hwang-Jae Lee, Hyeon-Youl

151 Abstract Author Index Lee, Joo-Han Lee, Joyce S...693, 1104 Lee, Jung-Sun Lee, Karim...74, 575 Lee, Kee-ho Lee, Kwan Hee...65, 547, 551 Lee, Kyuhyun Lee, Minhyung...268, 273, 943 Lee, Ryang Hwa...305, 1038 Lee, Sang K Lee, Sang-Jin...56 Lee, Seong-Wook...700, 1029 Lee, Solomon Lee, Soonhee...489, 492 Lee, Sung-il Lee, Suyeon Lee, Vivian Lee, Won-Il...835, 853, 1036 Lee, Yoen Kyung Lee, Young-Ju Lee, Young-Sook Lee, Zhenghong Leen, Ann M Leggiero, Eleonora...13 Lehenkari, P...53 Lehn, Pierre...207, 905 Lei, Yixiong Leibovitch, Ilan Lei-Butters, Diana C. M...385, 719, 848 Leja, Justyna Lejukole, Henry Y Lekmine, Fatima Lemoine, François Lemoine, Nick Lemp, Nathan A Lendlein, Andreas...304, 927 Leng, Xigang Lentacker, Ine Lenz, Jack...67 Leong, Kam W...734, 883 Leopold, Philip L...100, 242, 419, 1109 Leppänen, Pia Lerner, Seth P Lernmark, Ake...105, 106 Lesavre, N Lesch, Hanna P...307, 468, 785 Lesina, Eugenia Lesniak, Maciej S Leturcq, France Leung, Kathryn Leung, Kitty Lev, Dina Levy, Philippe Levy, Robert J...122, 123, 334 Levy, Ron Lewin, Alfred S...529, 983 Lewis, Danny H...170, 182 Lewis, David L Lewis, Mark G Lewis, Melissa A Lewis, Susanna M...4 Li, Anna A...521, 935 Li, Baozheng Li, Chang Long Li, Chengwen Li, Chuan-Yuan Li, Fang Li, Feng Li, Gang Li, Haitang , 1100 Li, Haiting Li, Hojun...7, 839 Li, Hong Li, Honghua Li, Hongwei...275, 360, 1025 Li, Hsiao Ping Li, James Li, Jianbin...138, 142 Li, Jing Li, Juan...138, 142, 955 Li, Linhong Li, Ming-Jie Li, Qiliang Li, Quanhai

152 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Li, Senlin Li, Shaudong...25 Li, Shawn Li, Sheng...129, 148, 153, 409 Li, Shirley...364, 563, 567 Li, Song Li, Tsai-ping Li, Weidong Li, Wenzhong...304, 927 Li, Xiao L...33, 621 Li, Xiaofei Li, Xiao-Kang...450, 684 Li, Xiaomiao...829, 846 Li, Xinyan Li, Xiong...56 Li, Ya-Chen Li, Yi...1, 413 Li, Ying Suet Li, Yong Fu Li, Zheng Li, Zong-Yi...36, 54, 200, 331, 1111 Liabeuf, Marie Liang, Zhong Liao, I.-Chien Lieber, Andre...36, 54, 200, 221, 244, 331, 378, 683, 1111 Lieberman, Jay R Liebold, Andreas...304, 927 Lievens, Joke Liimatainen, Timo Likar, Yuri Lim, Chae-Lyul Lim, Filip...535, 1040 Lim, Kwang-il Limbach, Keith...16, 148, 153 Limberis, Maria...8, 258, 415 Lin, Chang Xiu Lin, Chi-Chen Lin, Chung-Ren Lin, Feng...503, 504 Lin, Jianping...8, 255, 257, 366 Lin, Marie C...234, 609 Lin, Yukang...214, 370 Lindholm, Leif Lindner, Lars Lindtsedt, Sean Ling, Changchun Ling, Leona Link, Charles J Linnes, Michael P Linton, Gilda Lipschutz, Joshua H Lisboa, Bianca C...488, 554 Lisowski, Leszek Liu, An-Bang Liu, C Liu, Chien-Cheug Liu, Chunsheng...47 Liu, Chunyan...34, 174, 176, 177, 181, 252, 538, 758, 887 Liu, Dexi...929, 22 Liu, Fang Liu, Feng Liu, Gang Liu, Guei-Sheung Liu, Gumei...62 Liu, Hao Liu, Hongli...50 Liu, Jason B Liu, Jian Liu, Jianhua...2 Liu, Jilin Liu, Linda N...185, 431 Liu, Mingdong...70 Liu, Olga Liu, Pei-Qi Liu, Po-Ching Liu, Shanling Liu, Song...29 Liu, Te-Hui Liu, Xiang Liu, Xiang-Qin Liu, Xiaoming Liu, Xibao Liu, Xingge Liu, Yang

153 Abstract Author Index Liu, Yemin Liu, Yi-Lin Liu, Ying...36, 54, 200, 331, 1111 Liu, Yong...40, 115, 316, 827, 832, 852 Liu, You-Hong...56 Liu, Zhiqiang Lizonova, Alena Lo, Hsin Lung Lobos-González, Lorena Locci, Michela Lock, Martin Lockett, Linda J Loew, Rainer Logg, Christopher R...762, 796 Logroscino, Giandomenico Loh, Peng Y...286, 671 Lohse, Ansgar W Lombardo, Angelo Lombardo, Barbara Long, Carole A Long, Chun...133, 722, 850 Lonkar, Pallavi S Looney, Benjamin M Lopez, Patricia Lopez-Franco, Oscar...329, 330, 870 Loring, Jeanne Lorson, Chris L Lorson, Christian L Lotta, Jr, Louis T Lou, Howard...100, 419, 1109 Louboutin, J.-P...367, 988 Louboutin, Jean-Pierre...245, 526, 530, 532, 543, 682, 997, 1043, 1095 Low, Walter...109, 993, 1044 Lowenstein, P. R Lowenstein, Pedro R...34, 174, 176, 177, 181, 252, 538, 758, 887 Lu, Baogen Lu, Hui...3, 955 Lu, Lanxia Lu, Li...266, 577 Lu, Ning Lu, Pei Juan Lu, Qi L Lu, Qi Long...128, 701, 713 Lu, Xiang Lu, Yi Lu, Yuanqing...844, 1045 Lucarelli, Guido Lucas, Bart Lucca, A. F Lucia, Vanrell Luckay, A Ludwig, Christine Luetzow, Karola Lufino, Michele M. P Lui, Song Lui, Xingge Luigi, Naldini Luke, Jeremy Lulka, Hubert...204, 1042 Lundberg, Cecilia Lundin, Karin E Luo, Dan...906, 908 Luo, Meihui Luo, Min...457, 565 Luo, Rongcheng...40 Luo, Zhengyu...353, 974 Lupo Stanghellini, M. T Lupold, Shawn E Luthi-Carter, Ruth Lützow, Karola Luu, Yvonne...346, 909 Lux, Kerstin...75 Lyons, Gray R Ma, Congrong...4, 387 Ma, Guangyu Ma, Hong...328, 782, 838 Ma, Jiangzhong Ma, Jun Ma, Nan...304, 927 Ma, Wenqin Ma, Xiucui...26, 351, 650, 1065 Maatta, Antti I MacDonald, Robert C Mackay, Charles R

154 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index MacLeod, Sheena H MacNeil, Angus Maeda, Akito Maeda, Yasushi Maeder, Morgan...58 Maetzig, Tobias Maggioni, Daniela...37 Magnani, Z Magnusson, Maria K Maguire, Al M Maguire, Albert M...743, 745 Maguire, Henry Mah, Cathryn Mah, Cathryn S Mahadevan, Mahendran...40 Mahato, Ram I...715, 1027, 1031 Mahdy, Ayman E. M Mahller, Yonatan Y...394, 461, 608 Mähönen, Anssi J...307, 468 Maier, Dawn G Maier, Patrick...603, 634, 828 Maina, Njeri...829, 846, 855 Mainka, Astrid Maisner, Andrea Maitra, Amarnath Makarem, Maisam Mäkelä, Anna R Makhija, Sharmila...182, 868 Mäkinen, Petri I Makino, Elina R Malech, Harry L...322, 650, 768, 769 Malehorn, Matthew Malekzadeh, Reza Malgieri, A...64 Malik, Punam...772, 775 Malik, Saafan Mall, Christine I Manabe, Ichiro Mandel, Ronald J...25, 529 Mandfredsson, Fredric P...25 Mane, Viraj...406, 1083, 1088 Manfredsson, Fredric P Mangual, Viviana...687, 1021 Mann, Christopher J Manninen, Hannu Mano, Miguel...6 Mansfield, S. Gary Manthorpe, Marston Mantovani, Julie Mantwill, Klaus Mao, Hai-Quan Maples, Phillip B...141, 172, 382, 605, 614, 687, 1021, 1102 Marangoni, Francesco...247, 648 Marban, Eduardo...60 March, Keith L Marchesini, Sergio...96, 350 Marcovecchio, Paola Mardaleishvili, Konstantin Margison, Geoffrey P Marhofer, Juergen Maria Sol, Rodriguez-Pena Marieke, Bossink Marie-Turner, Anne Marigo, Valeria Marktel, Sarah Marquez, Ana...449, 452 Marsala, Martin Marsala, Silvia Marshall, John Martin, Douglas R Martin, Heather...31 Martin, Molly E Martin, Oana M Martín-Bermejo, M. Jesús...535, 1040 Martinez-Fong, Daniel Martinez-Rizo, Abril A Martinez-Rodriguez, Herminia Martins, Ines...57, 62 Martuza, Robert L Marusich, Elena I...560, 566, 568 Maruyama, Kazuo...491, 924 Mas, Alex Masago, Kayo Mas-Monteys, Alex

155 Abstract Author Index Masquelier, Anne-Marie Mastaglio, Sara...39 Mastrangelo, Michael A...30, 539 Masuda, Tomoya...894, 899 Masurier, Carole Matar, Majed M Matern, Dietrich Mathiesen, I Mathiesen, Iacob...161, 503, 504 Mathis, J. Michael...33, 621 Mathis, Michael J Matise, Ilze Matsukura, Makoto Matsumoto, Kazuya Matsumoto, Kunio Matsumura, Hajime Matsunaga, Akinao Matsuo, Keiko Matsushita, Saori Matsushita, Takasahi Matsushita, Takashi Matsushita, Yoko Matthews, Qiana L...437, 886 Matushita, Takashi Matusik, Robert J Matyakin, Evgeny Maucksch, Christof Mauleon, Itsaso Mauzy, Mary Jean Mavilio, Fulvio...238, 766, 1060 Maxwell, Christina R...543, 559 Maya, Ivan Maynard, Jonathan...150, 163, 168 Mays, Lauren Mazumder, M. A. Jafar Mazzoleni, Stefania Mc Niel, E. A McBride, Aaron...9 McBride, Jodi L...57 McCaffrey, Anton...580, 665, 1101 McCarty, Douglas M...97, 982 McChesney, Michael B McClard, Ronald W McCombs, Connie McConnell, Ruth McCormack, William M McCourtie, Anton S McCown, Thomas McCray, Paul B McCray, Jr, Paul B...675, 1004, 1007 Mccue, Scott McDonald, Cari J...47 McDonald, Donald M McEwan, Jean R McGarrity, Gerard J McHale, Anthony P McIntosh, Jenny H...103, 670 McIntosh, Tom J...906, 908 McIvor, R. Scott...24, 109, 220, 235, 339, 656, 686, 912,...993, 1044, 1070 McIvor, R. Scott C McKay, Tristan R...324, 416, 1013 McLachlan, Gerry...420, 1068 McLaren, Jay W McLauchlan, David L McLear, Julie McMahon, Jill McMahon, Ross...309, 312 McMahon, Stephen B McMenamin, Deirdre McNamara, Andrew R...32 McNulty, Joseph C McPherson, David D McVey, Duncan...153, 884, 891 McVey, John H...329, 857, 870 McVey, John J. H Mease, Philip J Mecham, Robert Medin, Jeffrey A...48 Medina, Maria Fe C Meechoovet, Hunsar B...561, 663 Mehrian-Shai, Ruty Mehta, Jawahar L...115, 316, 827 Meinking, Keith

156 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Melendez, J. Andres Mellon, Matthew J Melo, Vinicius Meltzer, Stephen J Mena, Mayra Menander, K. B...45 Menander, Kerstin Mendel, Matthew Mendell, Jerry R...136, 823, 1075, 1077 Meng, Cuixiang Meng, Qing-Hai Meng, Xiangdong Merchant, Milton J Merkel, Christian A Mermod, Nicholas...73 Meroni, Germana Merritt, J. Lawrence Merten, Otto Meseck, Marcia...49 Messer, Anne Messina, Emily L Messow, Diana Metzger, Mark Meulenberg, Janneke...85, 88, 348, 1056 Meuse, Leonard...134, 409, 721, 1022, 1078 Meyer, Johann...647, 1061 Meyer, Martin Meyers, Craig A...25 Meznarich, Norman A Miao, Carol H...20, 253, 959, 1087 Miao, Lv...463, 620 Miccio, Annarita...250, 651, 1060 Michaud, Annick Michelsen, Kathrin S Midoux, Patrick Miettinen, S...53 Miike, Teruhisha Mikheeva, Galina Mikkelsen, Jacob G...69, 338, 793, 1028 Miklavcic, Damijan Milano, Carmelo A Millar, Jamie Miller, A. D...418, 1009 Miller, A. Dusty Miller, Aaron M Miller, Andrew Miller, Daniel G...1 Miller, James R Miller, Jeffrey C...436, 777 Miller, Jim Millington-Ward, Sophia Millot, Loic Milsom, Michael D Milstone, Leonard Mimuro, Jun...359, 836 Min, Kyunghyun Min, SeokHong Mineno, Junichi...314, 315, 575 Mineshiba, Fumi Mingozzi, Federico...259, 843, 1056, 1085, 1089 Minn, H...45 Minoura, Arisa Mir, Lluis M...500, 918 Mirek, Hanna Mirolo, Massimiliano...238, 766 Misery, Laurent Mishina, Yuji Mishra, Anjali Miskin, James E...71 Misteli, Heidi Mitalipov, Shoukhrat M Mitani, Ko Mitchell, James B Mitomo, Katsuyuki Mitra, Koyel...67 Mitrophanous, Kyriacos A...71 Mitsui, Kaoru Mitsui, Masaru Mittal, Suresh K Miyake, Koichi Miyake, Noriko Miyake, Takashi Miyanohara, Atsushi Miyano-Kurosaki, Naoko...180,

157 Abstract Author Index Miyatake, Shin-ichi Mizuguchi, Hiroyuki...400, 669, 859, 861, 875, 882 Mizukami, Hiroaki...86, 108, 359, 761, 836 Mizuno, Masaaki Mizutani, Eiji Moal, Yohann D...71 Moayeri, Morvarid Mochizuki, Seibu Modlich, Ute...647, 770, 1061 Mody, Disha...33 Moehle, Erica A Moen, Robert C Moezzi, Lili Mohamadnejad, Mahdi Mohamed, Abdalla J Mohammad Alizadeh, Amir-Hooshang Moimas, Silvia Mok, Stephen Moldt, Brian...69, 338, 793, 1028 Molina, Gustavo C Moller, Pernille H...19, 500, 679, 914 Monahan, Paul E Mondkar, Sonali...181, 252, 887 Monkar, Sonali Monnat, Jr, Raymond...5, 269 Monni, O Monreal, Iñaki Monroe, W. Todd...930, 934 Montane, Joel Montefusco, Sandro Monteleone, G...64 Montforte, Joseph K Montgomery, Chyrstal L Montier, Tristan...207, 905 Montini, Eugenio...303, 350, 766, 1063 Montus, Marie Moore, Nicole M Moore, P. F Morabito, Paolo Moraes, Jane Z Moraes, Mauro P Morcos, Paul A Moreland, Rod Moreno-Flores, M. Teresa Moreno-Flores, Maria Teresa Morgan, Rick Mori, Keisuke Mori, Takeshi...511, 933, 1071 Mori, Yuriko Morianos, John Moriguchi, Rumiko Morishita, Ryuichi...534, 681, 992 Moritz, Thomas...246, 652 Morizono, Kouki Morozov, Viktor...61 Morral, Nuria Morris, John C Morris, Kevin V...278, 707 Morrissey, Anothony Morrow Buff, Scotty Mortelmans, Luc Moschou-Parara, Myrsini...8 Moser, Markus Moullier, Philippe Mountz, John Mouscadet, Jean Francois...73 Mudhakir, Diky Muehlebach, Michael D Mueller, Oliver J Muenzer, Joseph...93, 97, 824 Mufamadi, Steven M Mufazalov, Fagim Muhammad, A. K. M Muhammad, A. K. M. G...34, 177, 178, 181, 758 Muller, Ina Muller, Susanne Mullins, Charles...358, 964 Multack, Richard F Multhaup, Megan M Munden, Regina Muñoz-Brauning, Carlos Munroe, David...4 Munson, Keith...719, 724 Muppidi, Raghu

158 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Murakami, Miho Murakami, Nao Muramatsu, Shin-ichi Murata, Masashi...14 Murofushi, Yoshiteru Murphy, Samuel L...7, 839 Mussolino, Claudio...528, 533, 746, 826 Muthumani, Karuppiah Mutlu, Gokhan M Muul, Linda Muvarak, Nidal...256, 295, 297, 691 Muzyczka, Nicholas...25, 529 Muzyczka, Nick Myers, Gary D Naash, Muna I...397, 910 Nabel, Gary J...860, 891 Nagai, Ayako Nagai, Katsuyuki...446, 600 Nagai, Ryozo Nagasaki, Takeshi Nagasaki, Yukio Nagashima, Noriko Nagy, Tim R Nah, Hyun-Duck Naidoo, Tanusha Nakagaki, Noriaki Nakagami, Hironori Nakagawa, Aya Nakagawa, Shinsaku...875, 882 Nakai, Hiroyuki...4, 135, 387, 665, 1074 Nakai, Michio...14 Nakajima, Yoshiyuki...299, 958 Nakamura, Shin-ichiro Nakamura, Toshikazu...213, 534, 681 Nakanishi, Mahito...794, 1098 Nakanishi, Yoichi...787, 888 Nakano, Imaharu Nakashima, Hiroshi Nakashima, Kazuko Nakata, Masanori Nakatsuji, Norio Nakayama, Masaharu Nakayama, Toshinori Nalbantoglu, Josephine Naldini, Luigi...96, 247, 303, 321, 350, 377, 405, 423, 434,...766, 774, 1063, 1082, 1106 Namavari, Mohammad Nan, Zenhong Nandakumar, Satish Nantz, Michael H Nara, Yuko Naranjo, Araceli...663, 1051 Narfstrom, Kristina Narrow, Wade C...458, 539 Närvänen, Ale...393, 464 Nasu, Yasutomo Natarajan, Shobha Nathwani, Amit C...103, 670, 977 Natsume, Atsushi Naumann, Nora Navaratnarajah, Chanakha K Naylor, Stuart Nedeljkovic-Kurepa, Ana Neefe, John R Neff, Andreas...21 Neff, Tobias Negishi, Yoichi...480, 491, 924 Neglia, Simona Neilan, John Nelson, Angel Nelson, Angelique M Nelson, Joel B Nemunaitis, Gregory Nemunaitis, J. J...45, 1052 Nemunaitis, John...141, 172, 188, 222, 382, 443, 585, 605,...606, 614, 644, 687, 728, 1019, 1021, 1102 Nemunaitis, Michael...188, 443, 728 Nemunitais, John Neri, Margherita...96, 1035, 1106 Nettelbeck, Dirk M...441, 885 Neumann, Eberhard Neville, Chales M Nevin, Barry Newman, Joseph

159 Abstract Author Index Newton, Alexandra C Ng, Chee P Ng, P Ng, Philip...252, 417, 887, 964, 1016 Nguyen, Frederic M...25 Nguyen, Tien V Nguyen, Viet Ni, Shaoheng...36, 221 Nichols, Carmen N Nichols, Gina Nichols, W. S Nicklin, Stuart A...329, 330, 355, 857, 870 Nicosia, Alfredo Nicoud, Marjorie Nielsen, Troels T Nienaber, Jeffrey Nienhuis, Arthur...773, 806 Nierman, Melchior C Nietupski, Jennifer B Niidome, Takuro...480, 511, 520, 933, 1071 Niikura, Kenichi Nikhamin, Yan Nikolai, Bryan C Nimura, Keisuke Niranjan, A...55 Nishida, Hiroko Nishihira, Jun...401, 552 Nishikawa, Makiya...165, 343, 625 Nishikawa, Tomoyuki Nishimura, Ken Nishio, Takashi Nishiyama, Akiyo...135, 1074 Niskanen, Einari A...307, 467 Niu, Yunyun Nixon, Alan J Nnakamura, Takafumi Noguiez-Hellin, Patricia Noh, Moon Jong...65, 547, 551 Noh, Yoohun Nokisalmi, Petri Nomicos, Effie Nonoguchi, Naosuke Noorinayer, Babak Norberg, Johanna Norris, James...382, 591 Norton, James E Novelli, G...64 Noyes, Marcus B Nozawa, Iwao...450, 684 Nukaya, Ikuei...314, 315 Nunez-Alonso, Graciela...948, 1008, 1018 Nwanegbo, Edward C Nylander, Karen Oberg, Kjell O Brien, Timothy...114, 354, 1046 Ocaranza, Paula Ochiai, Hiroshi Ochiai, Takenori...190, 622 Ochoa, Laura Ochoa, M. Sofia Ochs, Hans D...253, 1087 O Connor, Timothy P...317, 979 Oda, Yusuke Odaka, Yoshinobu...33, 621 O Doherty, Aideen Odom, Guy O Donnell, Patricia...26, 650 O Driscoll, Caitriona M Oerlemans-Bergs, Carla J. L Oezgun, Numan O Gara, Peter Ogawa, Koichi Ogawa, Ryohei Oggiano, Leonardo Ogihara, Toshio...681, 992 Ogris, Manfred...495, 928, 1073 Oh, Sang Su Oh, Sangtaek Oh, Sung-Min Oh, Tae Keun Ohana, Patricia Ohashi, Kazuo...299, 958 Ohkawara, Tatsuya Ohlfest, J. R

160 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Ohno, Yuji Ohshima, Sachiko Ohshima, Shigeki Oikawa, Tatsuo Oishi, Jun Oishi, Motoi Oishi, Yumiko Oka, Kazuhiro...352, 358 Okada, Hideho...55 Okada, Michiyo Okada, Naoki Okada, Takashi...108, 135, 359, 761, 834, 1074 Okada, Yoshiaki Okadah, Kaori Okamoto, Hiroshi Okano, Sinji Okano, Teruo Okbinoglu, Tulin...66 Oker-Blom, Christian Oku, Naoto...231, 710 Okuyama, Hiromi Olagüe, Cristina Olaru, Alexandru Olivare, Simon Olivares, Eric C Oliver, Mueller...76 Olsen, John C Olsen, Philip...772, 799, 800 Olson, Erik...482, 771, 912, 1037 Olszewska, Malgorzata Olutunmbi, Femi O Mahony, Deirdre O Malley, Thomas...72, 104, 650 O Neill, Lee O Neill, Martin J Ong, Hooi T Ong, Lee-Lee...304, 927 Onimatsu, Hideki Ono, Fumiko Onodera, Shin...401, 552 Opolon, Paule O Reilly, Mary O Riordan, Catherine R Orme, Ann O Rourke, Donald M Ortiz-Lopez, Rocio Osada, Naoki Osaki, Mitsuhiko Osborn, Mark J...95 Osborne, William R...105, 106 Oscherwitz, Jon Oshima, Shigeki Oshimura, Mitsuo Ostberg, Julie R O Sullivan, Gerald C...502, 917, 919, 920 Otano, Itziar O Toole, Dermot Otsuki, Akihiro Otsuru, Satoru Ott, Marion G Ouchi, Masaaki...446, 763 Oupicky, David Oxendine, Ila...687, 1021 Ozasa, Shiro Ozawa, Keiya...85, 86, 108, 359, 761, 834, 836 Ozbas-Turan, Suna Ozturk, Ferhat Ozuer, Ali Pacak, Christina A...821, 990 Padilla-Rivas, Gerardo Paez-Cortez, Jesus Pagel, Mark Painter, Hazel Pal, Ranajit Palacios, Jose Alejandro...6 Palese, Peter Palfi, Arpad Palmer, Brent Palmer, D Palmer, Donna...252, 417, 887, 964, 1016 Palmieri, Lucia Pan, Chien-Hsiung Pan, Dao Pan, Weihong...706,

161 Abstract Author Index Panakanti, Ravikiran Panaroni, Cristina Pandey, Aseem Pane, Fabrizio Paneda, Astrid...362, 831 Pañedas, Astrid Panet, Amos Pang, Jijing Papanikolaou, Eleni Papapetrou, Eirini Pâques, Frédéric Paraskevakou, Georgia...51 Pareyn, Inge Pariente, Nonia Park, Choul Y Park, Dongsu Park, Gyu Park, Hye-Ran Park, Jeonghyun...489, 492 Park, Keerang...637, 643, 835 Park, Min-Tae Park, Paul...294, 1084 Park, Shay E Park, Soon-Hye Park, Steven Park, Yong Serk Parker, Alan L...329, 330, 870 Parker, Jack Parker, Kristin Parker, William B Parkinson, Rose M Parks, Robin J Parmentier, Laurie Parrilla, Claudio Parsons, David...416, 1005, 1012 Parvin, Most Nahid Pasceri, Peter Paschon, David...373, 777 Pasqualini, Renata Passini, Marco A Pastor, Eric J...87, 313 Pastore, Lucio...13, 876 Patel, Ami...149, 757 Patel, Manij Pattarini, Lucia...357, 980 Patterson, Noelle B...16, 148, 153 Paulk, Nicole...522, 840 Paun, Bogdan Pautke, Christoph...21 Pavar, Mahesh Pavlakis, George B Pavsel, Natasa Pearson, Daniel Peccatori, J Pecorini, Giovanni Pedersen, Darlene A Pedersen, Line...150, 168 Pedersen, Nina Peer, Dan Peeters, Liesbeth Peled, Michael Peltekian, Elise Peluso, Richard...57, 87, 313 Pence, Casey Peng, Baowei...20, 253, 1087 Peng, Kah-Whye Peng, Weidan...43 Pénicaud, Luc Perabo, Luca...75 Peranteau, William H...256, 295 Perea, Javier Pereboev, Alexander...33 Pereboeva, Larisa...15, 879 Pereira, Ulysse Perez, Aleida Perez, Christophe Perez, Elena E Perez-Alvarez, Ingrid Perlin, David Perna, S Péron, Jean-Marie Perricaudet, Michel Perrier, Michel...89 Perrin, Valerie

162 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Persons, Derek...248, 773, 806, 1107 Perumbeti, Anil Peshwa, Madhusudan V...185, 431 Pesonen, Sari Pestina, Pestina I Pestina, Tamara I Peter, Felsburg J Peters, Paul J Petersen, Bryon Peterson, Elise B...30 Peterson, Laura J...323, 424 Petrigliano, Frank Petrillo, Marco...826, 999 Petrus, Inge Petry, Harald Pfaffenzeller, Irene Pfeifer, Alexander...696, 804 Philipp, Alexander Piasecki, Julia Pichavant, Christophe Pichon, Chantal Pickles, Raymond J Pierce, Glenn F Pierre, Lorraine F Piggott, John...502, 917, 919, 920 Piguet, Francoise Pike, Lisa Ping, Gao Pinkert, Sandra...694, 847, 851 Pinto, Carlos...349, 352 Pipe, Steven W...406, 675, 955 Piret, James M...817, 818 Pittaluga, Stefania Pivirotto, Phil Plank, Christian...21, 928 Platt, Virginia M Plinkert, Peter Pluhar, G. E Podetz-Pedersen, Kelly...24, 235 Podetz-Pederson, Kelly M Podetz-Petersen, Kelly Podhajcer, Osvaldo L Podsakoff, Greg Poduturri, Jagat...33, 621 Poeschla, Eric M Poh, Chit Laa Poirier, Amy...990, 1054 Pola, Enrico Pola, Roberto Policastro, Lucía L Polisky, Barry...57, 699 Politi, Letterio S...377, 423 Poller, Wolfgang...694, 847, 851 Pollock, Raphael E Poluri, Anantha Lakshmi Polyak, Steven F Ponder, Katherine P...26, 72, 351, 650, 1065 Ponnazhagan, Selvarangan...192, 206, 549, 760 Ponomarev, Vladimir Pons, Jennifer Ponzoni, Maurilio...39, 250, 1063 Poon, Betty Poon, Ronnie T. P Pope, C. Earle Pope, Melissa A Popplewell, Leslie Porada, Christopher D...294, 1084 Porter, Andrew C. G Porteus, Matthew H...58 Porvasnik, Stacy...825, 983 Pos, Petra Postrach, Johannes Potter, Murray...521, 935, 947 Potter, Philip M Poulard, Karine Poulsen, Hans S Poulsen, Thomas T Poupiot, Jérôme Powell, Russell...193, 237 Power, John M Pownall, Scott Pradayrol, Lucien...204, 633, 1042, 1103 Praetorius, Mark Prata, Carla

163 Abstract Author Index Préat, Véronique Prentice, Howard M Press, Oliver W...214, 370 Preuss, Meredith A...124, 862 Price, Matthew...8 Prieto, Jesús...189, 362, 421, 469, 792, 831 Pringle, Ian A...420, 1008, 1015, 1018 Pritchard, David...54 Prockop, Darwin J...305, 1038, 1049 Provasi, E Pruett, Shondra M...58 Przybylowski, Mark Psatha, Nikoletta Pucci, Ferdinando Pugh, Jr, Edward N Pule, Martin...38 Puligadda, Uday Pulin, Andrey A...305, 1038 Pun, Suzie H...506, 737, 926 Puntel, M...758, 178 Puntel, Mariana...181, 252, 887 Puri, Raj K...51 Putt, David Puttaraju, M Qi, Senrong Qi, Yan-Ting Qian, Cheng Qian, Hu Sheng Qian, Kun Qiao, Chunping...138, 142 Qin, F. Xiao-Feng Qiu, Gang Qiu, JianPing...582, 597, 755 Qiu, Xingpin Qiu, Yujie...487, 1033 Qu, Guang...90, 91 Quaglino, Elena Quattrini, Angelo Quenneville, Simon Quezada, Valerie R Quintanilla, María Elena Quintarelli, Concetta...38 Quiroga, Jorge Raake, Philip...97 Rábano, Alberto...535, 1040 Rabkin, Samuel D Rabussay, Dietmar...157, 503, 504 Raccosta, Laura...37 Rached, Carlos Racz, Gabor Z...286, 671 Radrizzani, Marina...39 Radu, Antoneta...256, 295, 691 Radu, Antonetta Radu, Caius Rainelli, Cristina...37 Rainey, Kevin D Rajagopal, Ramesh Rajecki, M Rajecki, Maria Rajeshwari, Mehta R Raki, Mari Ralph, Gareth S...71 Ramakrishnan, S Ramesh, Rajagopal...197, 1097 Ramezani, Ali Ramirez-Correa, Genaro A...60 Rao, Donald...382, 614, 1021, 1102 Raoul, Cedric Raoul, Sylvie Rastegar, Mojgan Ratliff, Timothy L...44 Ratner, Buddy D Ratner, Nancy Rattmann, Ina...246, 652 Räty, Jani K...393, 464 Raubitschek, Andrew Raut, Sanj Ravet, Emmanuel...633, 1042 Rawlings, David J Rayford, Walter Razquin, Nerea Rebar, Edward J...373, 777 Rebuffat, Alexandre Recchia, Alessandra...238, 766,

164 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Reddy, P. Seshidhar Redinbo, Matthew R Reding, Mark T Rehman, Khaja K...678, 1093 Reichart, Bruno Reid, T Reid, Tony Reik, Andreas...373, 585 Reineke, Theresa M...505, 736 Reiser, Jakob...425, 531, 816 Reiter, Christopher D Reiter, Robert E Rekke, Belia Rembao-Bojorquez, Jesus D Remling, Leila...292, 563 Ren, Changchun Rentsendorj, Atan Reppen, Tom Resnick, Daniel Rethoré, Gildas Rettig, Garrett R...523, 524 Rex, Tonia S...745, 854 Reyes, Beverly A. S...526, 530, 532, 543, 682, 988, 997, 1095 Reyes, Morayma...113, 139, 970, 1110 Reyes del Valle, Jorge Rezende, Fernando C Rhim, Jung Woo...74 Rhodes, James Ribas, Antoni...34, 176, 374 Rice, Jennifer S Rice, Karen Rice, Kevin G...523, 524, 735 Richard, Isabelle Richard, Robert E Ricks, David M Riddell, Stanley Riekkinen, Mervi Rigolet, Aude Riley, James L Riley, Jonathan...77, 284, 677 Rinaldi, Paola Rininger, Joseph A Rinne, Johanna...465, 467 Ripberger, Maria C Riu, Efren...340, 341 Rivera, Angel A...437, 604, 621 Riviere, Christel Riviere, Isabelle Rizo, Liza Rizzo, Kathyrn A Robbins, Jeffrey Robbins, Paul D...196, 346, 546, 678, 1093 Robert, Donahue Roberts, James L Roberts, Laurel E Roberts, Michael L Robinson, Michael J Rock, Jeremy M Rockemann, Daniel D Rodgers, John Rodino-Klapac, Louise R...136, 1075 Rodrigues, Margret S Rodriguez, Carlos Rodriguez, Ester...864, 881 Rodriguez, Luis L Rodriguez, Ron Rodriguez, Ronald Rodriguez-Madoz, Juan R...189, 792 Roesch, Paula L Roessler, Blake...42 Roffler, Steve R...36, 584, 586 Rogers, Chantrice Rogers, Faye A...703, 708 Rogers, James Rognoni, Emanuel Rohmer, Stanimira Rojas-Martinez, Augusto Rolland, Alain...145, 756 Rols, Marie-Pierre Romero, Carol Romero, Norma Romero, Pedro Romero-Diaz, Viktor Roncarolo, Maria-Grazia...238, 247, 350, 648, 766,

165 Abstract Author Index Ronda, Jocelyn Ronkainen, Paula Rooney, C. M Rooney, Cliona M...38, 368 Roques, Caroline Rosas, Lucia E Roschier, Miia M Rose, John K Roselli, Emanuela A Rosen, Elliot D Rosenblat, Mira Rosenblum, Rachel Rosenfeld, Jeffrey...128, 479 Rosier-Montus, Marie Roskelley, Eric M Ross, Colin J. D...88 Rossi, Claudia...250, 651 Rossi, Gabriela R Rossi, John...557, 562, 563, 567, 570, 702, 1098, 1100 Roth, Jack A Roth, Justin...15, 879 Roth, Michael D Rousseau, Joel Routledge, Samantha Rovelli, Attilio Roy, Krishnendu...175, 932 Roy, Soumitra...155, 661 Rozema, David B Rubanyi, Gabor...221, 285 Rubinchik, Semyon Rubio-Zapata, Hector A Rudolph, Carsten...485, 913 Rudolph, Cornelia Rudy, John Rufer, Nathalie Ruhela, Dipali Ruiz, Juan Rupniewski, Igor Rusalov, Denis Russ, Verena Russell, Alan J Russell, David W...1, 413, 422, 771, 791, 1037 Russell, Stephen J...47, 51, 52, 764, 786 Russo, Vincenzo...37 Ruszniewski, Philippe Rutledge, Elizabeth A Ryser, Martin F Ryu, Ji-Hoon Saad, Antonio F Saayman, Sheena Sabatino, Denise...951, 1085 Sabo, Peter J...70 Sachdeva, Geetanjali Sacramento, Chester B Sadadcharam, Mira Saddekni, Souheil Sadelain, Michel...427, 583, 592, 645, 953 Sadilek, Martin Saeki, Yoshinaga...392, 394, 460, 608, 994 Saetrom, Pal Sahenk, Zarife...136, 1077 Saito, Izumu...14, 281 Sakaguchi, Masakiyo Sakai, Ryo...384, 612, 763 Sakata, Yoichi...359, 836 Sakiyama-Elbert, Shelly E Sakthivel, Bhuvana Sakurai, Fuminori...669, 859, 861, 875, 882 Sakurai, Haruna...875, 882 Sakurai, Kazuo Salazar, Adriana Sallberg, Matti Salmon, André Salo, Jarmo Salomoni, M Saltzman, Daniel...220, 686 Salva, Maja...422, 1037 Salvatore, Francesco Samal, Siba K Samia, Anna C. S Sampaolesi, Maurilio Samulski, R. Jude...3, 386, 389, 838, 842 Samuni, Yuval Sanchez, M

166 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Sanchez-Bonilla, Marilyn Sanders, Niek N...490, 507, 902, 1020, 1069 Sandhu, Arbansjust Sandoval, Ana...449, 452 Sands, Mark S...1 Sanges, Daniela Sangiuolo, F...64 Sankar, Uma Sano, Masayuki Santoni de Sio, Francesca...303, 1106 Santoro, A Santos, Elmer Sanvito, Francesca...250, 1063 Sapag, Amalia...680, 688 Sarkar, Fazlul Sarkar, Rita...951, 955, 960 Särkioja, M...53, 601, 617 Sarra, M...64 Sartori, Daniela Sartori, Serenella Sasaki, Erika Sasaki, Tomomi Sasaki, Yasuo Sastry, Lakshmi...306, 325 Sato, Ayako Sato, Fumiaki Sato, Makoto...193, 233, 237, 627 Sato, Motoki Sato, Naoyuki Sato, Susumu Satoh, Taku Satyamurthy, Nagichettiar Saul, Justin M...737, 926 Savoldo, B Savoldo, Barbara...38 Savontaus, Mikko...632, 635 Sawamura, Kaori Sawicki, Janet A...43 Sawtell, Nancy M Saxena, Romil Sayegh, Mohamed Scaramuzza, Samantha...247, 303, 648, 766 Schachern, Patricia A Schaefer, Caralee Schaefer, Hansjoerg Schaffer, Chris Schaffer, David...80, 807 Schaft, Niels Schaller, Laura Schambach, Axel...246, 327, 770, 805, 1041, 1061 Scharenberg, Andrew M Schaser, Thomas Schenkwein, Diana A. M Schepp-Berglind, Jennifer Scherman, Daniel...915, 940, 1079 Scheule, Ronald K...107, 720, 747, 1017 Schierer, Stephan Schlacterman, Alexander...7 Schleef, Martin Schlegelberger, Brigitte Schleifman, Erica B Schlesselman, James J Schmidt, Manfred...350, 240, 303, 1058, 1060, 1063 Schmitz, Joern E Schmoeckel, Michael Schnepp, Bruce...81, 151 Schoenly, Kimberly A Scholler, Nathalie Scholte, Bob J Schowalter, David B Schreifels, Jeffrey M...339, 482 Schroeder, Mark...51 Schuchman, Edward H Schuettrumpf, Joerg...2 Schüle, Silke Schuler, Gerold Schultz, Brian...113, 721 Schumacher, Ton Schwartz, Jamie D...100, 979 Schwartz, Justin Schwartz, Rachel...6 Schwarze, Ulrike Schwarzwaelder, Kerstin Schweizer, Matthias

167 Abstract Author Index Schwerdt, Alenka Sciascia, Sandra Scimeca, Michael S Score, Paul R Scott, Jeannine...9, 11, 659, 660, 664 Scotti, Giuseppe Scotti, Marina M Scrinivasin, Rahul...28 Sebestyen, Magdolna G Seebacher, Jan Seethammagari, Mamatha...171, 1113 Segal, David J...274, 288 Segawa, Hiroaki Seger, Reinhard Seiler, Michael...406, 1083, 1088 Sellner, Leopold Selves, Janick Semenova, Elena...16, 153 Sena-Esteves, Miguel Senesse, P Senina, Svetlana Senzer, Neil...172, 188, 222, 382, 443, 614, 644, 728, 1019, , 1050, 1102 Seo, Min Jeong...305, 1038 Seo, Su Jin Seol, Dai-Wu Seol, Wongi Seppo, Yla-Herttuala Seregin, Sergey S Sergi Sergi, Lucia...405, 1063, 1082 Sergio, Marchesini Serrano, Luis Sersa, Gregor Servida, P Sesenoglu-Laird, Ozge Sessa, Maria Settembre, Carmine...94 Sevin, Caroline Seymour, Len Shababi, Monir...749, 985 Shaffer, James Shahi, Payam Shaish, Aviv Shakeley, Misty Shalev, Moshe Shanker, Manish Sharif, Faisal Sharma, Anupriya Sharma, Anurag Sharma, Kamal Shashkova, Elena V Shayakhmetov, Dmitry M...863, 871 Sheard, Vicky Shen, Feng Shen, Heng-Chun Shen, Hongmei...302, 1108 Shen, Yuqiao...614, 1102 Shen, Zan Shen, Zhenping...20 Shenker, Larissa Sherk, Chris Shew, Chwen Y Shi, Min Shi, Qin Shiau, Ai-Li Shibata, Hiroaki Shibata, Masaru Shibata, Satoko Shihabuddin, Lamya S...31, 987 Shiiba, Haruka Shilkaitis, Anne Shilling, Chris J Shilling, Christopher J Shima, Midori Shimada, Hideaki Shimada, Kazuyuki Shimada, Masamitsu Shimada, Naohiko Shimada, Takashi...111, 251, 347 Shimada, Yutaka Shimamura, Munehisa Shimbo, Takashi Shimotaka, Kazunori Shimozato, Osamu

168 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Shin, Jin-Hong...135, 1074 Shin, Oh Kyo Shin, OhKyu...853, 1036 Shin, Won Shinkai, Seiji...401, 945 Shinohara, Mitsuru Shirakawa, Toshiro...631, 729 Shiraya, Suguru Shirayoshi, Yasuaki Shivakumar, Rama Shroll, Joshua Shu, Chengyi J Sia, Kian Chuan...463, 620 Siapati, Elena K Sibley, Don A...33 Sicam, Julie Sidhu, Maninder K Sidi, Abraham A Sidone, Brian...42 Siefring, Jamie Siegal, Gene P...604, 727, 868 Siegel, Donald...8 Sievers, Richard E Si-Hua, Mao Siler, Ulrich Silva-Platas, Christian I Simón, Diana...535, 1040 Simonson, Oscar E Sims, Stuart Sin, Sang-Joon Singh, Ankur Singh, Harjeet Singh, Mahender...245, 568, 1043 Sinigaglia, Milena Sinn, Patrick L...675, 783, 1004 Sipo, Isaac...694, 847 Sitia, Giovanni Siu, Karen Skele, Kristine L Skinner, Amy M Skog, Johan Skripchenko, Yelena Slagter-Jager, Jacoba G Slavin, S Slawin, Kevin M Slayton, William...829, 846 Sleeper, Meg Slepushkin, Vladimir Slobodkin, Gregory Smerdou, Cristian...189, 792 Smith, Annette N Smith, Bruce F...130, 722, 727 Smith, Carolina B Smith, Edvard C. I Smith, George-Wesley T Smith, Jeffrey S...12, 856 Smith, Julianne Smith, Katherine Smith, Tracy...199, 203, 475 Smits, Kristen Smolke, Christina D Snapper, Jolanda Snella, Elizabeth M...98, 996 Snoeys, Jan...99, 333, 676 Snyder, Richard O Soares, Luiz A. L Soares, Rafael Sobol, R. E...45 Sobol, Robert E Sobrevals, Luciano Soden, Declan M...502, 917, 919, 920 Söderholm, Jonas...161, 504 Sofer-Podesta, Carolina Soghomonyan, Suren Sohn, Joo-Hyuk Sokolic, Robert A Soleimani, Masoud Somanathan, Suryanarayan Somanthan, Suryanarayan Somia, Nikunj V...205, 235, 656 Son, Kang-Won Sonabend, Adam M Sondhi, Dolan...317, 748 Song, Antonius N

169 Abstract Author Index Song, Cunxian...123, 972 Song, Hyun Song, In Ho Song, Min-Sun Song, Shaochun...143, 1048 Song, Sihong...844, 1045, 1054 Song, Yuhu Sonntag, Susan Sonpavde, Guru Sorensen, Charlotte B Sorenson, Brent Soresen, Brent Sorscher, Eric J Soundara Manickam, Devika Souque, Anny...204, 1103 Sova, Pavel...54, 200, 244, 1111 Sowers, Anastasia L Spampanato, Carmine...94 Sparvero, L. J Spencer, Brian...98 Spencer, Carolyn T Spencer, David M...171, 214, 1113 Spencer, H. Trent...407, 408 Spencer, Jacqueline F Spencer, Lindo T Speth, Robert C Spier, Isabel Spitalieri, P...64 Spivak, Ryan M Spratt, S. Kaye...32, 270, 442 Springer, Matthew L Springfeld, Christoph Srivastava, Arun...388, 829, 846, 855 Srivastava, Kumar Stabban, Chris Staber, Patrick...57, 699 Stachler, Matthew D...78, 383, 725 Stacpoole, Peter Staecker, Hinrich Ståhl, Stefan Stamatoyannopoulos, George...244, 692, 778, 1062 Stamatoyannopoulos, John A...70 Staniforth, Vanisree Stapleton, Gary Starr, Renate Stastny, Michael Staunstrup, Nicklas H...69, 793 Steel, Jason C Steenssens, Laurette...8 Steer, Clifford J Stefaniak, Maureen E...16, 148, 153 Stefanski, Jolanta Stehle, Thilo Stein, Stefan Steinhoff, Gustav...304, 927 Steinkasserer, Alexander Steinman, Ralph M Steitz, Julia Stemberger, Axel...21 Stenman, U.-H Stepan, Tony Stewart, Jeff Stiefelhagen, Marius Stocker, Alice G Stoddard, Barry L Stoff-Khalili, Mariam A...437, 621 Stone, Daniel Stone, Marvin J Storb, Rainer F Storer, William A...17 Storm, Terry...5, 840 Storm, Theresa A...4, 59, 387, 693, 1104 Stover, Harald Strand, Sabina P Strauss, Bryan E Strauss, Robert...54, 200, 1111 Strayer, David S...245, 365, 367, 526, 530, 532, 543, 559,...560, 566, 568, 571, 576, 682, 873, 988, 997, 1043, 1095 Strayer, Marlene S...576, 873 Stripecke, Renata Stroes, Erik S Strong, Jim Strychor, Sandy...42 Styche, Alexis

170 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Su, Hua Su, Mei...67 Subbarayan, Murugessan Subbotin, Vladimir Suckau, Lennart...694, 847, 851 Sud, Reeteka...424, 799 Suda, Takeshi...22 Sueishi, Katsuo Suemori, Hirofumi Sugito, Takayuki Suita, Sachiyo Sullenger, Bruce A...211, 476 Sullivan, Layla...25 Sullivan, Sean M...271, 1070 Sumida, Michihiro Summey, Victoria Sumner-Jones, Stephanie G Sumners, Colin...275, 360, 1025 Sun, Baodong Sun, Jianmin Sun, Jing Sun, Junjiang Sun, Wenchao Sun, Wenchoa Sun, Xun Sung, Li-Yu...471, 550 Sung, Randall S Sung, Young C Suominen, Elina Surace, Enrico M...112, 528, 533, 826 Surace, Enrico Maria...94, 746 Surosky, Richard Susanto, Melisa T Sutherland, W...45 Sutton, Richard E Suzuki, Eisaku Suzuki, Keiichiro Suzuki, Masataka Suzuki, Nobuo Suzuki, Ryo...491, 924 Suzuki, Taketo Suzuki, Yuko Svahn, Mathias G Svenson, Amanda G Swanson, Debra...235, 656 Sweet, Ian R Symes, Julie...48 Szafran, April F Szczepek, Michal Szoka, Francis C Szoka, Jr, Francis C Szymanski, Paul Tabata, Ken-ichi Tabata, Toshiaki Tabata, Yasuhiko Tachibana, Katsuro Tafuro, Sabrina...120, 357 Tagami, Tatsuaki...493, 710 Tagawa, Masatoshi...622, 631 Taguchi, Tomoaki Tai, Alex Tai, Ming-Hong Tajiri, Tatsuro Tak, Paul P Takagawa, Junya Takahashi, Hidenori Takahashi, Hiroshi Takahashi, Tomoyuki Takahashi, Yuki Takahashi, Yuzuka Takaku, Hiroshi...180, 789 Takakura, Yoshinobu...165, 343, 625 Takano, Jun-ichiro Takata, Yuki Takatsy, Zsuzsanna Takayama, Koichi...787, 888 Takeda, Shin ichi...135, 1074 Takeda, Shuko Takeda, Yoichi Takei, Yoshifumi Takemoto, Seiji...165, 343 Takenaka, Atsushi...631, 729 Takesako, Kazutoh Takeshita, Daniela

171 Abstract Author Index Takeuchi, Daisuke Takiguchi, Masato Takino, Naomi Takizawa, Tomoko...491, 924 Taksir, Tatyana V...31 Tal, Kafri Tal, Reshef Talmage, Brown Tam, Philip Tam, Phillip Y Tamai, Katsuto...971, 1112 Tamaki, Yasuhiro Tambellini, Rodrigo Tamhane, Mayur C...399, 569 Tampier, Lutske Tan, Eng Lee Tan, Erdal Tan, May Chin Theresa Tan, Siyuan...32 Tanaka, Kazushi Tanaka, Motoyoshi...496, 685 Tanaka, Naoko I Tanaka, Nobumichi Tanaka, Noriaki...384, 600, 612, 763 Tanaka, Sakura Tanaka, Shigeru Tang, Qiushi Tang, Tong Tang, Yizhe...878, 886 Tang, Yucheng...150, 163, 168 Tangey, Mark Tani, Kenzaburo...657, 787 Tanimoto, Ryuta Tanioka, Yoshikuni Tannous, Bakhos...613, 779 Tao, Mi-Hua Tapscott, Stephen J Tarantal, Alice F...298, 398, 556 Tarkkanen, Maija Tashiro, Katsuhisa...859, 875, 882 Tateno, Chise...299, 958 Tatsumi, Kohei...299, 958 Tatsuta, Kyosuke Tayi, Venkata S...817, 818 Taylor, Brad N Taylor, Clare Taylor, Jason A Taymans, Jean-Marc Teissié, Justin Tellez, Joe ten Hagen, Timo L Teng, Qingshan...77, 284, 677 Tenhunen, M Tenhunen, Mikko...632, 639 Tennant, Lucinda Tennant, Peter Terao, Keiji...575, 836, 861 Terao, Shuji...631, 729 Ternovoi, Vladimir V Terpstra, Anton H. M Tessitore, Alessandra Tetz, Lauren Thaiss, Friedrich Thakur, Roopa Thattaliyath, Bijoy Theobald, Narda Thibodeau-Beganny, Stacey Thiry, Irina Thomale, Jurgen Thomas, Charlie...16, 153 Thompson, Eric Thompson, Timothy C Thompson, Jr, Donald F...17 Thomson, Kassandra Thony, Beat Thorne, Barbara A Thorne, Steve Thorpe, Robin Thrasher, Adrian...324, 416, 648, 1013, 1058, 1060 Thresher, Randy J Throm, Stacy Thyagarajan, Bhaskar Thys, Chantal Tian, Chaorui

172 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Tian, Jie...12 Tiboni, Francesca...250, 651 Tikhonenko, Maria O Tilesi, Francesca Timmermans, Eric J Tindall, Donald J Ting, Alice Y...78 Tiraby, Gérard Tittiger, Mindy...26, 351, 1065 Titus, Louisa F Tjelle, Torunn...161, 503, 504 Todo, Tomoki Toida, Riki Toivanen, Pyry I Toivonen, Raine Tolar, Jakub...95 Toma, S Toma, Salvatore...39 Tomimatsu, Nozomi Tomioka, Atsushi Tong, Alex...222, 380, 443, 585, 606, 614, 728, 1021, 1102 Tononi, Laura...96, 303, 350 Tordo, Noel Torgerson, Troy Torrisani, Jérôme Toso, Raffaella Toth, Karoly Towers, Greg Traas, Anne...26, 351 Traas, Jeremy Traktuev, Dmitry Tran, CoVi Tran, Kaylie Tran, Nhu...373, 585 Tran, Truong A Tranquart, François Tranter, Mike Trauner, Dirk Traversari, Catia...37 Traversari, K Trehin, Rachel Treleaven, Christopher...31 Tremblay, Jacques P , 1080, 1081 Tremethick, David J Trifari, Sara Trinh, Alex Trobridge, Grant D...66, 239, 424, 562, 772, 800 Tron, Victor A Trossen, Alycia A Trucco, Massimo Trygg, Cyndi Tsai, Anthony Y Tsuchiya, Hiroyuki Tsuji, Atsushi...450, 684 Tsujii, Hirohiko Tsukamoto, Taiji Tsurkan, Lyudmila Tsuruta, Yuko Tu, Ying Tuohy, Gearoid Tupling, Terry...105, 106 Turchetto, L Turkki, Vesa P Turner, Lorianne S Turner, Roy James Turpeinen, Sanna Turunen, Mikko P Tuschong, Laura M...243, 249, 771 Tuve, Sebastian...36 Twisk, Jaap...85, 88, 348, 831, 977 Tyler, Matthew A Tyr, Olena Uchibori, Ryosuke...359, 761 Uchida, Yuji Uchino, Junji Uchino, Makoto Uchiyama, Masayuki Ueda, Yasuji...184, 190, 381 Uehara, Shuichiro Uemura, Hirotsugu...496, 685 Ueno, Shinji Uesugi, Kentaro Ugai, Hideyo...872, 878, 880 Ugen, Kenneth...18,

173 Abstract Author Index Ulasov, Ilya V Ungari, Silvia...96 Unger, Gretchen M Ungerechts, Guy Unimo, Yukimo Uno, Atsushi Uno, Futoshi Urabe, Akihiro...353, 974 Urabe, Masashi...359, 85, 86, 108, 761, 836 Urara, Yasuo Urata, Yasuo...446, 600, 612, 763 Urban, Nicole...54, 200, 589, 1111 Urbinati, Fabrizia...766, 775 Urnov, Fyodor D...321, 426, 777 Utoguchi, Naoki Vaessen, Stefan F. C Vahakangas, Elisa Vahanian, Nicholas Vaikunth, Sachin S Vaillend, Cyrille Valentine, John F Valentino, Leonard A Valentino, Michael Valero, Valderez B Vallant, Molly Valluripalli, Vinod K Valtolina, V van Amersfoort, Edwin S...88 van Beusechem, Victor W...445, 733 van Bockstaele, Elisabeth J...526, 530, 532, 682, 997, 1095 Van Craeyveld, Eline...99, 676 van de Kasteele, Willeke van den Doel, Petra B Van Den Haute, Chris van der Aa, Marieke A. E. M van der Laan, Luc J van der Wegen, Pascal van Deventer, Sander...85, 88, 348, 831 Van Geet, Chris van Marion, Ingrid van Til, Niek P van Zeeburg, Hester J. T Vandenberghe, Luk H...8, 254, 258, 366, 415, 723, 743, 822, 826, 854 Vandenbroucke, Roosmarijn E...507, 1069 VandenDriessche, Thierry...300, 646 Vandeputte, Nele Vanderberghe, Luk H VanDongen, Case VanEcho, D...45 Vangala, Rajani K Vanhoorelbeke, Karen Vanni, Holly VanOosten, Rebecca L VanRoey, Melinda VanRooijen, Nico...34 Vansant, Gordon A Varathalingam, Anusha , 1018 Varenika, Vanja Vargesse, Chandra Vårum, Kjell M Vasko, Ann-Jeanette Vasko, Michael Vassilopoulos, George Vaughan, Andrew E Vaughan, Erin E Vazquez-Juarez, Jose L Velasco, Marco Veldman, Robert Jan Veldwijk, Marlon R...649, 828 Venneri, Mary Anna Vera, Juan F...38 Verakis, Thomas Verma, Inder M...98 Vernejoul, Fabienne Verstegen, Monique M. A Vervoordeldonk, Margriet J. B. M Vetter, Roland Vidal, Samuel J Vider, Jelena Vigant, Frederic Vigen, Karl K Viggeswarapu, Manjula Vigne, Emmanuelle Vihinen-Ranta, Maija...465,

174 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Viholainen, Jenni I Vilalta, Adrian...145, 756 Viles, Kristi D Villa, Anna...247, 648 Villamizar, Nestor R Villegas, Leah R Vincent, Karen Virella-Lowell, Isabel L Virk, Mandeep Virkkunen, Pekka Vishwanath, Reena P Visigalli, Ilaria...96 Visser, Trudi P Vite, Charles...26 Vitolo, Joseph M Vockley, Gerard Vogler, Carole...1 Vöhringer, Sabine...10 Voit, Thomas Vojtech, Lucia Volna, Petra Volpers, Christoph von Degenfeld, Georges von Kalle, Christof...240, 303, 1058, 1063 Von Kalle, Christoff von Laer, Dorothee...557, 562 Vongpunsawad, Sompong Vorhies, John S Voss, Tyler T Voutetakis, Antonis Vrecenak, Jesse...131, 290 Vrettou, Christina Waddington, Simon...23, 324, 329, 330, 344, 416, 870, 989, 1013 Wadell, Göran Wade-Martins, Richard...23, 391 Wadkins, Randy M Waehler, Reinhard Wagemaker, Gerard Wagenblatt, Agnes Wagner, Ernst...495, 928, 1073 Wagner, Jamie...663, 1051 Wagner, Ralf Wagstaff, Kylie M...510, 740 Wakabayashi, Toshihiko Wakayama, Teruhiko Wakefield, Darren H Waldron, Lanaya...105, 106 Wales, Samantha Q Wallraven, Gladice Walsh, Christopher E...79 Walsh, Melanie Walter, Glenn A Walton, Nathaniel A Wang, Bing...138, 142 Wang, Chung-Huei K Wang, Danher Wang, Daniel Wang, Daren Wang, Grace Y...463, 620 Wang, Guoqing Wang, He Wang, Hongjie...244, 683 Wang, Huijie Wang, Hui-jie Wang, Jianbin...426, 436 Wang, Jianghua Wang, Jianxiang Wang, Jinhui...3, 955 Wang, Jinjuan Wang, Lei Wang, Lijun...92 Wang, Lili...255, 257, 743, 1086 Wang, Lora...693, 1104 Wang, Minghui...604, 868, 880 Wang, Nathaniel...426, 436 Wang, Pei Hsueh Wang, Peirong...413, 1037 Wang, Peiyin Wang, Ping...26, 650 Wang, Rui Wang, Tung-Hung Wang, Tza-Huei Wang, Weiwei Wang, Xiaomin...694,

175 Abstract Author Index Wang, Xicai Wang, Xiuli Wang, Yaohe Wang, Yingbing Wang, Yu...50 Wang, Yun Wang, Zaixing Wang, Zejing Wang, Zhongya...5 Wanjalla, Celestine Ward, Peter...79 Ware, Carol...422, 799, 1037 Washer, Sue Washizawa, Kentaro...795, 1010 Watanabe, Masaki Watanabe, Masami Watanabe, Yuichi...384, 600, 612, 763 Watkins, Simon C Wawrousek, Eric F...61 Wawrowsky, Kolja Waxman, David J Webster, Keith A Webster, Nikolas Wechuck, James...32, 986, 1000 Weger, Stefan Wegner, Nicholas J Wei, David S Wei, Zhangyong...745, 819, 854 Weinberg, Marco S Weiner, David B...146, 147, 162, 280, 751, 754 Weinhardt, Stephan Weiss, Deborah Weiss, Heidi Weisser-Thomas, Jutta Weissleder, Ralph Weitzman, Matthew D...6 Welte, Karl H Wen, Chih-Chun Wen, Shan Wendell, Steve K Wenk, Martin L Wenz, Frederik...603, 634, 649, 828 Wenzel, Daniela Wetzel, J. Denise Weyh, Thomas Whalen, Robert G...81 Wheeler, Michael D White, Michelle Whitley, Chester B...109, 802, 993, 1044 Wicke, Daniel...647, 770 Wicker, Jason A Widera, Georg Widmaier, Moritz Wielgosz, Matthew Wierdl, Monika Wilber, Andrew C Wilcox, David A Wiles, Siouxsie Wilke, Nicole Willer, Mette...41 Williams, Brenda Williams, David A...246, 652 Williams, Ivan Williams, James Williams, Jon P Williams, Richard D...44 Williams, Venus J Willmon, Candice Wilson, James M...8, 155, 254, 255, 257, 258, 366, 415, 574, 661,...723, 743, 755, 819, 822, 826, 1086 Wilson, Jim M Wilton, Steve D...63 Winnik, Francoise Wisse, Eddie Witek, Rafal P Witte, John F Witte, Owen N Witte, Thomas H Witting, Scott R Wohlfahrt, Martin E Wold, William S. M...588, 668 Wolf, Daniel A Wolfe, Darren...32, 310, 986, 1000 Wolfe, John H...27,

176 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Wolfe, Scot A Wolff, James E Wolff, Jon A...144, 1066 Wolff, Thomas Wolfrum, Nina Wong, Fay E Wong, So C Wong, Suet-Ping Wonganan, Piyanuch...13, 42 Woo, Savio L. C...49, 963 Woo, You-Sub Wood, Brent L Wood, Matthew J. A Woodard, Lauren E...337, 484, 957 Wooddell, Christine I Woraratanadharm, Jan Worden, Andrew Worgall, Stefan Work, Lorraine M Worsham1, Nicole Wortmann, Andreas...10 Wright, Christine...663, 1051 Wright, Fraser...745, 843, 1085 Wright, J. Fraser...90, 91, 432, 525 Wu, Bo Wu, Chao-Liang Wu, Chu-Chiao Wu, Di Wu, Hongju...437, 878, 886 Wu, Jian Wu, Jianqiang Wu, Lan...49 Wu, Lily...193, 233, 237, 627, 938 Wu, Ping Wu, Qi Wu, Robert Wu, Xiaolin...4, 243 Wulff, Holger Wurdinger, Thomas Wursthorn, Karsten...522, 840 Xagorari, Angeliki Xavier, Anguela M Xavier, Frederico Xia, Weiya Xiao, Weidong...3, 837, 955 Xiao, Wei-zhong Xiao, Xiao...138, 142, 955, 1093 Xie, Jing...3 Xie, Litao Xie, Xiaoming Xin, Meng Xing, Nancy Xiong, W...178, 758 Xiong, Weidong...252, 887 Xu, Feng Xu, Hui...59, 340 Xu, Li...81 Xu, Lingfei Xu, Ruian Xu, Ximing Xu, Yaqin Xu, Zhili...12, 856 Yabe, Idalia...884, 891 Yada, Toshihiko Yaghoubi, Shahriar S...596, 1051 Yagi, Naoto Yagi, Nobuhiro Yakumaru, Kousuke Yam, Priscilla...364, 563 Yamada, Masahisa Yamada, Shigeru Yamada, Tohru Yamaguchi, Kazuki Yamaguchi, Masaya Yamaguchi, Tomoko Yamamoto, Masato...195, 872 Yamaoka, Ayumi Yamaoka, Kiyoshi Yamashita, Shuzo Yamauchi, Masahiro Yamazaki, Naoshi Yamazaki, Takehiko Yamoutpoor, Farnaz Yamoutpour, Farnaz

177 Abstract Author Index Yan, Jian...162, 751 Yan, Ziying...385, 719, 848 Yanay, Ofer Yáñez-Muñoz, Rafael J...989, 1058 Yang, Guang Yang, Guanhua Yang, Ji Sun Yang, Jian Yang, Jun...284, 531, 677 Yang, Ning-Sun...154, 156, 164, 689 Yang, PingAr Yang, Qifeng Yang, Seung W Yang, Sherry W Yang, Tony Yang, Wen...266, 577 Yang, Wendy...31 Yang, Zhen Fan Yang, Zhonghui Yannaki, Evangelia Yant, Stephen R Yao, Feng Yao, Hong Yao, Jing...306, 308 Yao, Shuyuan Yasumura, Eduardo Yata, Tomoya Yayama, Katsutoshi Ye, Guo-jie Ye, Jianqin Ye, Peiqing...959, 1087 Ye, Zhaoyang...715, 1027 Yee, Jiing-Kuan...364, 563 Yeghiazarians, Yerem Yeh, Raymond Yeh, Yi-Chun Yen, Meng-Chi Yendluri, Satya Yeomans, David C Yew, Margaret Yew, Nelson S...342, 747, 1008 Yi, Youngsuk...65, 547, 551 Yin, HaiFang Ying, Ying...76 Yip, Ping Yip, Vivian...65, 551 Ylä-Herttuala, Seppo...210, 307, 393, 464, 467, 468, 1030 Ylikomi, T...53 Ylostalo, Joni...305, 1049 Ynag, Jing Yockman, James W...900, 1024 Yoder, Mervin C Yokoi, Katsutoshi Yokoo, Tomoko Yokosawa, Hideyoshi Yokota, Junichi Yonemisu, Yoshikazu Yonemitsu, Yoshikazu...184, 190, 787 Yoneyama, Yasuo Yoo, G. H...45 Yoo, Hyoung-Rea Yoo, Ji-Young Yoon, A-rum Yoon, Changshin...489, 492 Yoon, Hanna Yoon, Jaehyung...65, 551 Yoon, Nam-Kyung...74 Yoon, Sung Ok Yoshida, Jun Yoshida, Kimiko Yoshida, L Yoshida, Teruhiko Yoshihara, Chieko Yoshikawa, Kenichi Yoshimura, Naoki Yoshino, Toko Yoshioka, Akira...299, 958 Yoshioka, Hirofumi Yoshizaki, Mariko Yoshizato, Katsutoshi...299, 958 You, Changxuan...40 You, Hong Young, J. D Young, Leah C

178 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Abstract Author Index Young, Sarah P Yu, Gwang Sig...943, 946 Yu, Hong Yu, Hsiu Hui...156, 164 Yu, Hui Yu, Jie Yu, Julie Yu, Qi Yu, Seung Shin...314, 575 Yuasa, Katsutoshi Yue, Yongping...133, 722, 850 Yuen, Carrie H Yuichi Iida, Yuichi Yun, Chae-Ok...223, 228, 593, 615, 638, 640 Yun, Lillian...65, 551 Yun, Sung-Tae Zabala, Maider Zabner, Joseph...83, 335, 809 Zacchigna, Serena...120, 357, 980 Zaia, John...364, 563, 567 Zajac, Allison Zak, Roman Zali, Mohammad-Reza Zali, Narges Zamboni, William C...42 Zang, Weidong Zanjani, Esmail D...294, 1084 Zannis, Vassilis I Zanta-Boussif, Maria-Antonietta Zarzuela, Alexander Zaupa, Cecile M Zeichhardt, Heinz Zeiss, Caroline Zeithaml, Brian...328, 782, 838 Zelenaia, Olga...432, 525, 745 Zeller, Jens W...603, 634 Zeller, W. Jens...649, 828, 1059 Zentilin, Lorena...6, 120, 357, 980 Zern, Mark A Zhang, Chun Zhang, Guofeng Zhang, H. Steve...32, 776 Zhang, Haiping Zhang, Haoyue Zhang, Jane Zhang, Jian Zhang, Jun Zhang, L Zhang, Lei Zhang, Liang N Zhang, Ling Zhang, Liqun Zhang, Ming Zhang, Song-Nam Zhang, Steve H Zhang, Su Zhang, Ting Zhang, Wei Zhang, Weitian Zhang, Xiao-Bing Zhang, Xichen...199, 475 Zhang, Xu Zhang, Yanhong Zhang, Yan-Ping Zhang, Yi Zhang, Yu-An...443, 585, 728 Zhang, Yulong...385, 848 Zhang, Yunhan Zhang, Zhe Zhao, Huifen Zhao, Na...487, 1033 Zhao, Weihong...829, 846, 855 Zhao, Xingbo Zhao, Yuan Zheng, Changyu...318, 671, 841, 877 Zhi, Yan Zhong, Li...388, 829, 846, 855 Zhong, Zhiyuan...900, 1024 Zhou, Jiehua Zhou, Jingmin...90, 91 Zhou, Qing Zhou, Shangzhen...955, 1085, 1089 Zhou, Yuanyue Zhu, Fuxiang

179 Abstract Author Index Zhu, Hua Zhu, Jiangao Zhu, Liwei...487, 1033 Zhu, Yunxiang Zhu, Zeng B...733, 868 Zhuo, Jiacai...260, 263, 264 Ziady, Assem G Zibert, John R Ziegler, Robin J...107, 987 Ziegler, Steven Zillmer, Allen...128, 479 Zimmermann, Marcel Zimmermann, Martina Zingale, Anna...303, 774 Zingone, Adriana Zinn, Kurt R Zintchenko, Arkadi...495, 928 Zirk, Aaron F Zoet, Yvonne Zojaji, Homayoun Zollman, Paula...47 Zolotukhin, Sergei...102, 388, 829 Zoltick, Philip...131, 256, 290, 295, 297, 410, 691 Zou, Shaomin Zsak, Aniko Zsak, Lazlo Zuber, Mohammed Zuo, Yu...417,

180 Final Program ASGT 10th Annual Meeting Washington, USA May 30 - June 3, 2007 Notes 179

181 Notes 180

182

183