ASGT Annual Meeting

Program

ASGT 6th Annual Meeting
June 4-8, 2003, Washington, D.C.
Jointly sponsored by Baylor College of Medicine, Houston, Texas

Program Schedule

Thursday, June 5
7:00 am - 5:00 pm	Attendee Registration Open
8:00 am - 9:00 am	George Stamatoyannopoulos Lecture Marriott Ballroom Chair Donald B. Kohn, MD Speaker Gary Felsenfeld, PhD - Chromatin Boundaries and the Regulation of Gene Expression Excellence in Research Awards for Students and Post Doctoral Fellows Announced
9:00 am - 9:15 am	Break
9:15 am - 10:45 am	Workshops
	WS210 - Cancer: Replicating Vectors for Cancer Therapy Salon 2/3 Chair Stephen J. Russell, MD, PhD Speakers Stephen J. Russell, MD, PhD - Engineering Measles for Oncolytic Virotherapy Patrick W.K. Lee, PhD - The Molecular Basis of Reovirus Oncolysis Nori Kasahara, MD, PhD - Safety Considerations and Therapeutic Potential of Replication-Competent Retrovirus Vectors: Shifting the Risk vs. Benefit Paradigm David H. Kirn, MD - Replication-Selective Oncolytic Virotherapy with DNA Viruses: A Novel Therapeutic Platform for Cancer James Markert, MD - Engineered HSV-1 for Malignant Glioma John G. Hay, MD - Spread of Replicating Adenovirus in Established Tumors
	WS211 - Cardiovascular: New Concepts in Vascular Wall Modification Using a Gene Therapy Approach Cotillion South Chair Lawrence C. Chan, MD Speakers Lawrence C. Chan, MD - Gene Therapy for Dyslipidemia and for Diabetes Mellitus Larry Shears, MD - Effect of iNOS on Cardiac Allograft Vasculopathy Barry J. Byrne, MD, PhD - Targeting of AAV Vectors: Strategies for Cardiac and Vascular Gene Transfer Douglas Losordo, MD - VEGF Gene Therapy to Prevent Coronary Restenosis
	WS212 - Genetic Diseases: Genetic Disorders of the Eye Maryland Room Chair Jean Bennett, MD, PhD Speakers Alberto Auricchio, MD - AAV Vectors for Retinal Gene Transfer Jean Bennett, MD, PhD - Gene Therapy for Leber Congenital Amaurosis - Predictions Based on Studies in Murine and Canine Models John G. Flannery, PhD - Gene Therapy for Usher Syndrome Bill Hauswirth, PhD - AAV-Vectored Therapy for Neovascular Retinal Disease J. Timothy Stout, MD, PhD - Gene Therapy for Ocular Proliferative Disease
	WS213 - Hemopoietic: Marking Studies Virginia Room Chair Claudio Bordignon, MD Speakers Claudio Bordignon, MD - Multi-Lineage Stable Engraftment After Stem Cell Gene Therapy Combined With Mild Conditioning Cynthia Dunbar, MD - Large Animal Models Hans-Peter Kiem, MD - In Vivo Selection of Hematopoietic Stem Cells in a Large Animal Model Christof von Kalle, MD - Insertion Site Analysis in the Large Animal Model Jan Nolta, PhD - Xenograft Models to Study Human Stem Cell Biology and Gene Transfer
	WS214 - Industrial Liaison: Best Practice Approaches to Achieving GMP Manufacturing in Academia and Industry Washington Room 2 Chair Barrie Carter, PhD Speakers Richard O. Snyder, PhD - Recombinant Adeno-Associated Viral Vector Manufacturing Shawn L. Gallagher, MS - Adenoviral Manufacturing: Key Considerations in Finalizing CMC Content for a Biologics Licnese Application (BLA) Larry Couture, PhD - Plasmid DNA Manufacturing in a Multi-Product Manufacturing Facility Andy Lin, PhD - Manufacturing of Gvax®, GM-CSF Gene Modified Cancer Vaccine
	WS215 - Infectious Disease: Practical Application of RNA Interference Wilson Room Chair John J. Rossi, PhD Speakers Mark A. Kay, MD PhD - RNA Interference for the Treatment of Human Viral Hepatitis Roger J. Pomerantz, MD, FACP - RNA Interference and HIV-1 Entry Frederic D. Bushman, PhD - RNA Interference to Block Retroviral Infection David L. Lewis, PhD - In Vivo Delivery of Synthetic siRNA
	WS216 - Neural Disorders: Cellular Therapies for Neurological Disorders Washington Room 5/6 Chair Evan Snyder, MD, PhD Speakers Ron McKay, PhD - Using Embryonic Stem Cells to Understand and Treat Neurological Disorders Clive Svendsen, PhD - GDNF Secreting Human Neural Stem Cells for Parkinson's Disease and ALS Steven Goldman, MD, PhD - Gene-Therapeutic Induction of Neurogenesis From Endogenous Progenitors in the Adult CNS Alberto Martinez-Serrano, PhD - Human Neural Stem Cells for Neurodegenerative Diseases Evan Snyder, MD, PhD - Neural Stem Cell-Mediated Cross-Talk May Provide Therapeutic Opportunities
	WS217 - Non-Viral Vectors: Novel Non-Viral Vectors and Formulations for Gene Transfer Washington Room 4 Co-Chairs Kevin G. Rice, PhD Nancy Smyth Templeton, PhD Speakers David M. Lynn, PhD - Cationic Polymers and Macromolecular Assemblies for Gene Delivery Sung Wan Kim, PhD - Polymer-Based Local Therapeutic Gene Delivery Lei Zhang, PhD - Electroporation-Enhanced In Vivo DNA Delivery for Gene Therapy and DNA Vaccines Ronald K. Scheule, PhD - Catheter-Based Delivery of pDNA to Rabbit Liver Ralph W. Paul, PhD - Development and Characterization of a Lipid-Polycation-DNA (LPD) Lipopolyplex Formulation for Systemic Gene Delivery
	WS218 - Respiratory Tract: Approaches for Lung Gene Therapy Washington Room 1 Co-Chairs Eric W.F.W. Alton, MD, FRCP Joseph Zabner, MD Speakers John A. Chiorini, PhD - Use of AAV4 and AAV5 Vectors for Gene Transfer to the Lung Pamela Davis, MD, PhD - Molecular Conjugates for Accessing Airway Epithelium David A. Dean, PhD - Electroporation-Mediated Gene Transfer to the Lung Stephen Hyde, PhD - Identification of Cell Types Transfected by Viral and Non-Viral Vectors in the Lung Uta Griesenbach, PhD - Gene Silencing Strategies in the Airways
	WS219 - Viral: Viral Vectors and Innate Immunity Cotillion Ballroom North Chair Marshall S. Horwitz, MD Speakers E. Antonio Chiocca, MD, PhD - Innate Responses to HSV Vectors and Oncolytic Viruses Maria A. Croyle, PhD - Biochemical Modification of Viral Vectors to Reduce the Innate Immune Response William Wold, PhD - Adenovirus Oncolytic Vectors that Overexpress the Adenovirus Death Protein (ADP) Marshall S. Horwitz, MD - Adenovirus Immunoregulatory Genes for the Treatment of Autoimmune Diabetes
10:00 am - Noon	Poster Setup by Authors
10:45 am - 11:00 am	Break
11:00 am - 12:30 pm	Workshops
	WS220 - Cancer: Imaging Gene Expression Salon 2/3 Chair Sanjiv Gambhir, MD, PhD Speakers Sanjiv Gambhir, MD, PhD - Multimodality Molecular Imaging of Gene Therapy Kurt R. Zinn, DVM, PhD - High Resolution SPECT/CT Imaging of Genetic Reporters in Rodents Juri G. Gelovani, MD, PhD - Monitoring Biodistribution, Delivery and Expression of Genes by Different Viral and Non-Viral Vectors James Basilion, PhD - MR Imaging of Gene Expression
	WS221 - Cardiovascular: Gene Therapy for Myocardial Ischemia and Heart Failure Cotillion Ballroom South Chair Anthony Rosenzweig, MD Speakers Anthony Rosenzweig, MD - Targeting Cardiac Dysfunction Through Gene Transfer Todd K. Rosengart, MD - Angiogenic Therapy and Cellular Cardiomyoplasty in the Treatment of Heart Failure H. Bradley Nuss, PhD - Probing Cardiac Excitability by Gene Transfer Walter J. Koch, PhD - Genetic Manipulation of the beta-Adrenergic Receptor Kinase in Heart Failure
	WS222 - Genetic Diseases: Gene Therapy for Lysosomal Storage Disease and Disorders Maryland Room Chair Mark Haskins, VMD, PhD Speakers Mark Haskins, VMD, PhD - Gene Therapy in Large Animal Models Chester B. Whitley, PhD, MD - Progress and Challenges Toward Gene Therapy for Mucopolysaccharidosis Disease Seng H. Cheng, PhD - Prospects for AAV-mediated Gene Therapy of Fabry Disease Hee Kyung Jin, DVM, PhD - Intracerebral Mesenchymal Stem Cell Gene Therapy in the Niemann-Pick Disease Mouse
	WS223 - Industrial Liaison: Non-Clinical Toxicology Studies in Support of Licensure for Gene Therapies Workshop Recap/Report Washington Room 2 Chair Parris Burd, PhD Speakers Parris Burd, PhD - Brief Review of the Rationale and Need for the Meeting Anne M. Pilaro, PhD - General Overview of the Meeting Beth Hutchins, PhD - Breakout Session Reports: Adenovirus Maritza C. McIntyre, PhD - Breakout Session Reports: AAV Douglas J. Jolly, PhD - Breakout Session Reports: Retrovirus and Lentivirus Nancy Markovitz, PhD - Breakout Session Reports: Herpes
	WS224 - Musculo-Skeletal: Clinical Trials and Musculo-Skeletal Diseases Salon 1 Chair Olivier Danos, PhD Speakers Olivier Danos, PhD - Gene and Cell-based Approaches for Engineering and Repairing the Skeletal Muscle Serge Braun, PharmD, PhD - Results of Phase I Clinical Trial of Plasmid-dystroption Intramuscular Administration into Duchenne and Bedror Dystrophy Patients Scott D. Boden, MD - Gene Therapy for Bone Formation - Working Toward Clinical Trials Christopher Evans, PhD - Clinical Trials in the Gene Therapy of Arthritis
	WS225 - Neural Disorders: Dissecting Neurological Function through Gene Transfer Washington 5/6 Chair Martha C. Bohn, PhD Speakers Krys Bankiewicz, MD, PhD - In Vivo Imaging of AAV-mediated Gene Transfer in CNS Martha C. Bohn, PhD - Tet-regulatable rAAV Vectors for Neurotrophic Factor Gene Delivery to the CNS Beverly L. Davidson, PhD - Silencing Dominant Disease Genes By RNAi Nicole Deglon, PhD - Intraneural Aggregates, Striatal Dysfunction and Behavioral Deficits Following Lentiviral Overexpression of Mutated Huntington in Rats and Non-Human Primates
	WS226 - Non-Viral Vectors: Pre-Clinical Studies with Synthetic Gene Delivery Vectors Washington Room 4 Co-Chairs Richard Heller, PhD Dieter Gruenert, PhD Speakers Esther H. Chang, PhD - A Systemic, Tumor-targeting, Liposomal Delivery System Emmanuel Dauty, PhD - Intracellular Delivery of Nanomatic DNA Particles into Tumor Cells via the Folate Receptor Daniel Scherman, PhD - Targeted Plasmid Delivery Loree Heller, PhD - Electrically-Mediated Delivery of Plasmid DNA to Tumors Peter M. Hobart, PhD - Designing Safe and Efficacious Plasmid Based Vectors
	WS227 - Respiratory Tract: Planning and Executing a Gene Therapy Trial for the Respiratory Tract Washington 1 Chair Ronald G. Crystal, MD Speakers Arthur L. Beaudet, MD - Reduced Inflammation and Improved Airway Expression in Mice Using Helper-Dependant Adenoviral Terence Flotte, MD - Recombinant Adeno-Associated Virus (rAAV) Vector-Mediated Gene Therapy for Alpha-1 Antitrupsin Deficiency Steven M. Albelda, MD - Adenoviral-Based Gene Therapy for Mesothelioma: The Long and Winding Road Richard Moss, MD - Aerosol Transgene Delivery to the Respiratory Tract: The tgAAVCF Experience
	WS228 - Viral: New Viral Technologies Cotillion Ballroom North Chair Matthew Weitzman, PhD Speakers Matthew Weitzman, PhD - Selecting Targeted AAV Vectors From a Library That Displays Random Peptides R. Jude Samulski, PhD - AAV Tropism and In Vivo Transduction James M. Wilson, MD, PhD - Novel Primate DNA Viruses Matthias Gromeier, MD - Molecular Mechanism of Tumor-Specific Replication of Oncolytic Poliovirus Recombinants Samuel D. Rabkin, PhD - Oncolytic Herpes Simplex Virus Vectors
	WS229 - Vectors for Functional Genomics Wilson Room Co-Chairs Xandra Breakefield, PhD Yoshinaga Saeki, MD, PhD Speakers Kazuma Tomizuka, PhD - The Use of Human Artificial Chromosome Vectors for Genetic Modification of Mammalian Genome Joseph Glorioso, PhD - HSV Vectors and Gene Function Studies Richard Wade-Martins, DPhil - The iBAC: A High Capacity Infectious Vector System for Functional Studies of Genomic DNA Panayiotis Ioannou, PhD - Development of Intact Genomic Loci from the Human Genome Project for Therapeutic Applications
Noon - 7:00 pm	Poster Viewing
12:30 pm - 1:30 pm	Meet-the-Expert Lunches The Meet-the-Expert sessions are designed to provide an opportunity for participants to interact with experts and discuss topics related to their research. A lunch will be served during the program, which will begin at 12:30 pm and last for approximately one hour. The Meet-the-Expert sessions require tickets for admission. Tickets for these sessions are not included in the regular registration fees to attend the meeting. Ushers will be collecting tickets at the door of each session. Tickets are available at the registration desk. Please note that space is limited.
	ME230 - Genome Editing - Can It Become Gene Therapy? Harding Room Michael Blaese, MD
	ME231 - Advantages and Limitations of Gene Transfer into Peripheral Blood Lymphocytes vs. Hematopoetic Stem/Progenitor Cells Coolidge Room Claudio Bordignon, MD
	ME232 - Cardiovascular Gene Therapy Hoover Room David A. Dichek, MD
	ME233 - Design and Application of Herpes Virus Vectors in Gene Therapy McKinley Room Joseph Glorioso, PhD
	ME234 - Gene Therapy for Lysosomal Storage Disease Using Animal Models Park Tower Room 8212 Mark Haskins, VMD, PhD
	ME235 - Gene Therapy as a Jigsaw Puzzle: Experience from Putting Together Meaningful Gene Therapy Clinical Trials Park Tower Room 8216 Douglas J. Jolly, PhD
	ME236 - Gene Transfer Strategies for Treating Hemophilia, Hepatitis and Other Hepatic Disorders Park Tower Room 8219 Mark A. Kay, MD, PhD
1:45 pm - 4:15 pm	Simultaneous Oral Presentations
	OA240 - Therapeutic Opportunities and Safety Considerations Marriott Ballroom Salon 2/3 Moderators Christopher Baum, MD Robert G. Hawley, PhD (Abstracts #1 - #10)
	OA241 - Host Interactions and Cancer Therapy Maryland Room Moderators Erik S. Falck-Pedersen, PhD Dan Von Seggern, PhD (Abstracts #11 - #20)
	OA242 - Naked DNA: Methods Virginia Room Moderators Dexi Liu, PhD Kevin G. Rice, PhD (Abstracts #21 - #28)
	OA243 - Transduction Biology and Applications Marriott Ballroom Salon 1 Moderators John F. Engelhardt, PhD Selvarangan Ponnazhagan, PhD (Abstract #29 - #38)
	OA244 - Cancer Apoptosis Cotillion Ballroom North Moderators Isabella Atencio, PhD James S. Norris, PhD (Abstracts #39 - #43)
	OA245 - Cancer Vaccines Cotillion Ballroom North Moderators Estuardo Aguilar-Cordova, PhD Laura K. Aguilar, MD, PhD (Abstracts #44 - #48)
	OA246 - Gene Regulation: New Technology Washington Room 4 Moderators Katie M. Binley, PhD Casey C. Case, PhD (Abstracts #49 - #53)
	OA247 - Cardiovascular II Washington Room 4 Moderators Masahiko Hoshijima, MD, PhD Cathryn S. Mah, PhD (Abstracts #54 - #58)
	OA248 - Cancer Targeted Gene Therapy III Cotillion Ballroom South Moderators E. Antonio Chiocca, MD, PhD Pedro Lowenstein, MD, PhD (Abstracts #59 - #63)
	OA249 - New Approaches to Gene Therapy for Hemophilia Cotillion Ballroom South Moderators Terence Flotte, MD Markus Grompe, MD (Abstracts #64 - #68)
3:00 pm - 7:00 pm	Exhibit Hall Open
	Poster Session I Poster Session, Exhibits and Networking Reception Halls B/C RNA Virus Vectors I (Abstracts #69 through #98) Biology of Transduction (Abstracts #99 through #121) Targeting Ad Vectors and Host Response (Abstracts #122 through #159) Naked DNA: Methods (Abstracts #160 through #191) Genetic and Metabolic Diseases: Part I (Abstracts #192 through #221) Gene Therapy for the Nervous System I (Abstracts #222 through #247) Gene Therapy of Muscle Diseases (Abstracts #248 through #270) Novel Approaches for Hematopoietic Gene Therapy (Abstracts #271 through #293) Cancer Immunotherapy (Abstracts #294 through #318) Cancer Targeted Gene Therapy I (Abstracts #319 through #347) Gene Regulation: In Vivo Applications (Abstracts #348 through #379)