Program  
ASGT 8th Annual Meeting
June 1-5, 2005
 ·  America's Center, St. Louis, MO
Jointly sponsored by Stanford University School of Medicine, Stanford, California
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Program Schedule

(This program is subject to updates/changes.)

Thursday, June 2, 2005

George Stamatoyannopoulos Lecture 200
8:00 am – 9:00 am

Room: America’s Ballroom

Chair:
Mark A. Kay, MD, PhD

Speaker:
Thomas Tuschl, PhD
Mechanisms of siRNA- and miRNA-guided Gene Regulation in Mammals

 

Presentation of Excellence in Research Awards


Break
9:00 am - 9:15 am

Workshops
9:15 am - 10:45 am

Workshop 210
Cancer: Optimization of T-Cell Gene Therapy
Room: 123/127
Chair:
Laurence JN Cooper, MD, PhD
Speakers:
John C. Morris, MD - Role of Interleutem-15 in T cell Development and Function
Laurence JN Cooper, MD, PhD - Redirected Specifity of T cells - Improving Their Potential
Steven M. Albelda, MD - Augmenting T-cell Trafficking to Tumors
Bruce L. Levine, PhD - Cancer: Optimization of T-Cell Gene Therapy
Francesco M. Marincola, MD - Gene Profiling of Immune Responses Against Tumors

Workshop 211
Gene-Based Vaccines: New Developments for Genetic Vaccines
Room: 120/124
Chair:
David B. Weiner, PhD
Speakers:
Richard Heller, PhD - Electroporation Mediated Immunotherapy of Solid Tumors: Preclinical Results and Initiation of Clinical Study
Leaf Huang, PhD - LPD Nanoparticles: A Gene Vector Turned into a Potent Vaccine Carrier
David B. Weiner, PhD - Expanding the Potency and Breadth of the Immune Responses Induced by DNA Vaccines
Britta Elisabeth Wahren, MD, PhD - Means to Induce Mucosal Immunity in HIV Genetic Immunization
Ning-Sun Yang, PhD - Developing Multiple Viral Protein Genes and Virus-like Particles as Gene-based Vaccines

Workshop 212
Hemopoietic: Hemopoietic Stem Cell Gene Therapy
Room: Ferrara Theater
Chair:
Jan Nolta, PhD
Speakers:
Carolyn Wilson, PhD - Addressing Safety Concerns for Hematopoietic Stem Cell Gene Therapy - A FDA Perspective
Manuel Grez, PhD - Correction of Chronic Granulomatous Disease by Gene Therapy
Marina Cavazzana-Calvo, MD, PhD - Clinical and Biological Results from the SCID-XI Gene Therapy Trial
Stanton L. Gerson, MD - In Vivo Stem Cell Selection without Myeloablation
Alessandro Aiuti, MD, PhD - Gene Therapy of ADA-deficient SCID

Workshop 213
Immunology of Gene Therapy: Organ Specific Immunity
Room: 260/264
Chair:
Roland W. Herzog, PhD
Speakers:
Pedro Lowenstein, MD, PhD - Immune Responses to Viral Vectors in the Central Nervous System
John Iacomini, PhD - Reshaping the Immune System Using Gene Therapy
Roland W. Herzog, PhD - In Vivo Gene Transfer to Liver and Muscle; Two Organs, Two Different Stories
Hans-Peter Kiem, MD - Immune Responses to Gene-modified Hematopoietic Cells

Workshop 214
Industrial Liaison: Pre-Clinical Issues in Gene Therapy

Room: 263/267
Chair:
Douglas J. Jolly, PhD
Speakers:
Rebecca Sheets, PhD - Pre-clinical Development of Adenovirus-vectored HIV Vaccine Candidate
Joy Cavagnaro, PhD, DABT, RAC - Optimizing Design and Analysis of Preclinical Development Programs
Boro Dropulic, PhD - Clinical Translation of a Lentiviral Vector for the Treatment of HIV/AIDS
Peter K. Working, PhD, DABT - Preclinical Strategies: Taking an “Armed” Oncolytic Adenovirus to the Clinic

Workshop 215
Programs of Excellence in Gene Therapy
Room: 230/231
Chair:
Sonia I. Skarlatos, PhD
Speakers:
Sonia I. Skarlatos, PhD - PEGT Overview
Haig H. Kazazian, Jr., MD - Preclinical Gene Therapy of Hemophilia A with New AAV Serotypes
Ronald G. Crystal, MD - Gene therapy for Alpha 1-antitrypsin Deficiency with Intrapleural Administration of Novel AAV Serotypes
Barry London, MD, PhD - Gene Therapy During LVAD Support: Can We Fix the Broken Heart?
Stanley Riddell, MD - Regulating T cells for Immunotherapy by Gene Insertion
Richard A. Knazek, MD - The National Gene Vector Laboratories - Production, Toxicology, Repository, Clonality Testing and Long-Term Follow Up

Workshop 216
Respiratory Tract: What’s New in Animal Models for Lung Gene Therapy
Room: 265/266
Chair:
Stephen Hyde, PhD
Speakers:
Bruce Bunnell, PhD - Mouse or Monkey: What is the FDA Looking for in Pre-clinical Data?
Alice F. Tarantal, PhD - Monkey Model for Fetal Intrapulmonary Gene Transfer
John F. Engelhardt, PhD - Are Men Mice or Pigs? A Lung Gene Therapy and Cell Biology Perspective
Uta Griesenbach, PhD - FABp-CF Knockout Mice as Models for CFTR Gene Transfer
Gerry McLachlan, PhD - Evaluation Non-viral Gene Transfer in the Ovine Lung

Workshop 217
Viral: Key Design Issues for Oncolytic Vectors
Room: 240 Complex
Chair:
Samuel D. Rabkin, PhD
Speakers:
David L. Bartlett, MD - Options for Creating Mutant Poxviruses for Oncolytic Therapy
Noriyuki Kasahara, MD, PhD - Building a Better Mouse Virus: Design Strategies to Improve Therapeutic Efficacy and Safety of Replication-competent Retrovirus (RCR) Vectors for Cancer Gene Therapy
John C. Bell, PhD - VSV as an Oncolytic Agent
Stephen J. Russell, MD, PhD - Retargeting Attachment and Entry of Oncolytic Viruses
William S. M. Wold, PhD - Oncolytic Adenovirus Vectors that Overexpress ADP
Samuel D. Rabkin, PhD - Oncolytic Herpes Simplex Virus (HSV) Vectors

Break
10:45 am - 11:00 am

Workshops
11:00 am - 12:30 pm

Workshop 220
Cardiovascular Gene Therapy: From Bench to Bedside

Room: 120/124
Co-Chairs:
Anthony Rosenzweig, MD
Roger J. Hajjar, MD
Speakers:
Albert J. Sinusas, MD - Imaging of Angiogenesis and Gene Therapy In Vivo
R. Jude Samulski, PhD - Biological Nanoparticles: The Next Generation of Efficient Delivery Reagents
David Kaye, FRACP, PhD, FACC - Percutaneous Approaches to Cardiac Gene Transfer
Roger J. Hajjar, MD - The Road from Physiological Observation to Gene Therapy in Patients with Heart Failure

Workshop 221
Clinical and Regulatory Affairs: Cell and Gene Based Cancer Vaccines
Room: 263/267
Chair:
Dale Ando, MD
Speakers:
Karin Jooss, PhD - Combination Therapy of a GM-CSF Secreting Tumor Cell Vaccine and anti-CTLA-4: Preclinical Evaluation and Tox Studies
Drew Pardoll, MD, PhD - Targeting Immune Responses to Sites of Cancer Using Engineered Viruses and Bacteria
Stephen J. Russell, MD, PhD - Noninvasive Expression Monitoring of Oncolytic Viruses in Phase I Clinical Trials

Workshop 222
Genetic Diseases: Focus on the Metabolic Diseases of the Liver
Room: 265/266
Chair:
Jayanta Roy-Chowdhury, MD
Speakers:
Markus Grompe, MD - Strategies to Select Hepatocytes In Vivo
Cary Harding, MD - Gene and Cell-mediated Therapeutic Approaches to Phenyckeonuria
Jayanta Roy-Chowdhury, MD - SV40-based Vectors for Metabolic Diseases of the Liver and Beyond
Brendan Lee, MD, PhD - Helper-dependent Adenoviral Gene Therapy for Metabolic Liver Diseases

Workshop 223
Infectious Disease: Innate Immunity and Vector Function
Room: 123/127
Co-Chairs:
Bruce L. Levine, PhD
John A. Zaia, MD
Speakers:
Hildegund CJ Ertl, MD - Innate Immunity to Adenovirus
Bryan Williams, PhD - Synergistic Activation of Innate Immunity by Double-stranded RNA and CpG DNA
Bruce E. Torbett, PhD, MSPH - Are Viral-based Vectors and Their Payloads Seen as Pathogens?
Hua Yu, PhD - Stat3 Targeting and Immunity

Workshop 224
Musculo-Skeletal: Myogenic Stem Cells and Regeneration
Room: 230/231
Co-Chairs:
Jeffrey Chamberlain, PhD
Shin’ichi Takeda, MD, PhD
Speakers:
Shin’ichi Takeda, MD, PhD - Contribution of CD31-negative/CD45-negative Side Population Cells to Skeletal Muscle Regeneration
Morayma Reyes, MD, PhD - Muscle Multipotent Adult Progenitor Cells (MAPC): Their Origin, Role and Clinical Potential in Muscle Disease
Stephen D. Hauschka, PhD - Regulatory Cassette Design for Optimal Expression in Skeletal and Cardiac Muscle Following In Vivo and Ex Vivo Gene Delivery
Thomas A. Rando, MD, PhD - Molecular Control of Muscle Stem Cell Activity
Johnny Huard, PhD - Gene Therapy Based on Muscle-derived Stem Cells: Potential for Tissue Regeneration and Repair
Emanuela Gussoni, PhD - Human Muscle Progenitor Cell Quiescence Maintained by Elevated BMP4 Expression

Workshop 225
Neural Disorders: Gene Therapy and Behavior
Room: Ferrara Theater
Chair:
Pedro Lowenstein, MD, PhD
Speakers:
Mary Bartlett Bunge, PhD - Using Gene Therapy to Improve Repair of the Injured Spinal Cord
William J. Bowers, PhD - Functional Assessment of Gene-based A² Immunotherapy in a Mouse Model of Alzheimer’s Disease
James B. Uney, PhD - Practical Approaches to Using Viral Vectors Provisional to Study the Molecular Basis of Behavioral Function
Michael Kaplitt, MD, PhD - Gene Therapy to Alter Sexual Behavior
Xandra Breakefield, PhD - HSV Amplicon Vector Delivery of Gonadotropin Releasing Hormone Gene (13 kb) to Hypothalamus Restores Estrous Activity in Hypogonadal Mice

Workshop 226
Nonviral: New Approaches in Nonviral Gene Delivery
Room: 260/264
Co-Chairs:
Stephen Hart, PhD
Leaf Huang, PhD
Speakers:
Esther H. Chang, PhD - Tumor Targeting Nanodelivery Systems: Expanding the Potential for Cancer Therapy and Diagnosis
Mark E. Davis, PhD - A Systems Approach to the Design and Development of Non-Viral Delivery Systems
Muna Naash, PhD - DNA Nanoparticles for Non-Viral Ocular Gene Transfer
Lonnie D. Shea, PhD - Controlled Release Systems for Non-viral Vectors
Alexander V. Kabanov, PhD - Transcriptional Activation of Gene Expression by Pluronic
Ian MacLachlan, PhD - Clinical Pharmacology of Stable Plasmid Lipid Particles for Systemic Administration

Workshop 227
Special: The Clinical Trial Data on adp53 Gene Therapy of Cancer
Room: 240 Complex
Co-Chairs:
James S. Norris, PhD
Helen E. Heslop, MD
Speakers:
Jack A. Roth, MD - Adp53 Clinical Trials
Dmitry Gabrilovich, MD, PhD - Immunotherapy of Cancer with Dendritic Cell Based p53 Vaccine
Yongsong Guan, MD - Transcatheter Hepatic Arterial Chemoembolization and Gendicine Combined in Treatment of Advanced Hepatocellular Carcinoma (HCC)

Workshop 228
Special: Skin Gene Therapy
Room: 261/262
Chair:
Ulrich R. Hengge, MD
Speakers:
Ulrich R. Hengge, MD - Naked DNA: From Basics to Application
Ernst Wagner, PhD - Bioresponsive Deshielding of Targeted DNA Polyplexes
Lorne Taichman, MD, MSc, PhD - Long-term Systemic Delivery From Genetically Modified Human Skin

Meet-the-Investigator Lunch Sessions
12:45 pm - 1:45 pm

Meet-the-Investigator 230
Room: Washington Avenue A
Chair:
Jeffrey Chamberlain, PhD - Systemic Delivery of Genes to Muscle Using rAAV

Meet-the-Investigator 231
Room: Washington Avenue B
Chair:
Carl H. June, MD - Translational Lessons on T Cell Gene Transfer

Meet-the-Investigator 232
Room: Washington Avenue C
Chair:
Donald B. Kohn, MD - Gene Therapy Using Hematopoietic Stem Cells

Meet-the-Investigator 233
Room: Washington Avenue E
Chair:
Gerry McLachlan, PhD - Can Animal Models Predict Clinical Efficacy of Respiratory Gene Transfer?

Meet-the-Investigator 234
Room: Washington Avenue F
Chair:
Maria Grazia Roncarolo, MD - Is Immune Response to Transgenes a Major Hurdle to Successful Gene Therapy?

Simultaneous Oral Abstract Sessions
2:00 pm - 4:30 pm

Oral Abstract Session 240
RNA Virus Vectors: Gene Transfer and Gene Expression

Room: 260/264
Co-Chairs:
Stephen J. Russell, MD, PhD
John Tisdale, MD
(Abstracts #1 - #10)

Oral Abstract Session 241
AAV Vectors: Vector Biolog
y
Room: 240 Complex
Co-Chairs:
Hiroyuki Nakai, MD, PhD
John F. Engelhardt, PhD
(Abstracts #11 - #20)

Oral Abstract Session 242
Adenovirus Vectors: Vector Technology
Room: Ferrara Theater
Co-Chairs:
Kohnosuke Mitani, PhD
Michael J. Imperiale, PhD
(Abstracts #21 - #30)

Oral Abstract Session 243
Inborn Errors of Metabolism - Stem Cells and Blood Disorders
Room: 120/124
Co-Chairs:
Barry J. Byrne, MD PhD
Mark S. Sands, PhD
(Abstracts #31 - #40)

Oral Abstract Session 244
Cardiac Gene Transfer
Room: 123/127
Chair: Federica Del Monte, MD, PhD
(Abstracts #41 - #50)

Oral Abstract Session 245
Neurodegenerative Diseases
Room: 263/267
Co-Chairs:
David J. Fink, MD
Martha C. Bohn, PhD
(Abstracts #51 - #60)

Oral Abstract Session 246
Gene Therapy for Infections and Vaccines
Room: 230/231
Co-Chairs:
Boro Dropulic, PhD
Bruce E. Torbett, PhD, MSPH
(Abstracts #61 - #68)

Oral Abstract Session 247
Cancer-Targeted Gene Therapy: Non Adenoviral Targeting of Vectors and Non Viral Vectors
Room: 265/266
Co-Chairs:
Ronald G. Crystal, MD
Albert B. Deisseroth, MD, PhD
(Abstracts #69 - #78)

Oral Abstract Session 248
Gene Regulation: Keeping Genes On and Turning Them Off
Room: 261/262
Co-Chairs:
Punam Malik, MD
Brian P. Sorrentino, MD
(Abstracts #79 - #88)

Poster Session I
4:00 pm - 7:30 pm
Hall 2

RNA Virus Vectors: Gene Expression
(Abstracts #89 through #109; and Abstract #822)

AAV Vectors: Disease Applications
(Abstracts #110 through #136)

Adenovirus Vectors: General Biology and Host Response
(Abstracts #137 through #154)

"Other" DNA Virus Vectors
(Abstracts #155 through #175)

Naked DNA Gene Transfer: Chromosomal, Integration, Delivery Techniques
(Abstracts #176 through #195)

Molecular Conjugates
(Abstracts #196 through #231)

Inborn Errors of Metabolism: Systemic and Storage Diseases
(Abstracts #232 through #250)

Muscle and Connective Tissue: Gene Therapy for Muscular Dystrophy
(Abstracts #251 through #266)

Cancer - Immunotherapy: Genetically Modified Antigen Presenting Cells and Adjuncts
(Abstracts #267 through #285)

Cancer-Apoptosis and Suicide
(Abstracts #286 through #309)

Cancer - Targeted Gene Therapy: Targeting of Non-Viral Vectors
(Abstracts #310 through #329; Abstract # 328 has been moved to Poster Session III)

Hematologic - Disease Models
(Abstracts #330 through #349)

Gene Therapy Approaches to Pulmonary Disease
(Abstracts #350 through #367)

Gene Regulation: Gene Targeting and RNA
(Abstracts #368 through #385)