Program Schedule

(This program is subject to updates/changes.)

• Wednesday, May 31
• Thursday, June 1
• Friday, June 2
• Saturday, June 3
• Sunday, June 4

Friday, June 2

Meet-the-Investigator Breakfast 300
6:45 am - 7:45 am
Room: 347
Speaker:
Katherine Ponder, MD
Gene Therapy for Mucopolysaccharidosis

Meet-the-Investigator Breakfast 301
6:45 am - 7:45 am
Room: 348
Speaker:
Savio L.C. Woo, PhD
Climbing the Academic Ladder in Gene and Cell Medicine

Scientific Symposium 310
8:00 am - 10:00 am
Gene Based Vaccines: Novel Prime Boost Regimens
Room: 327/329
Chair: Philip R. Johnson, MD

Speakers:
David T. Curiel, MD, PhD
Targeted Adenoviral Vectors for Dendritic Cell Transduction

John B. Haanen, MD
Short-Interval DNA Vaccination by Tattooing

James M. Wilson, MD, PhD
Nonhuman Adenovirus and AAV Vector Vaccine

Scientific Symposium 311
8:00 am - 10:00 am
Gene Therapy for Genetic Diseases: Treatment of Dominant Genetic Diseases
Room: 318/320
Chair: Beverly L. Davidson, PhD

Speakers:
Mimoun Azzouz, PhD
Treatment of Familial ALS Using Lentiviral Mediated Silencing of Mutant SODI

Seppo Yla-Herttuala, MD PhD
ApoB100 and LDL Receptor: Possibilities to Treat Hyperlipidemias with Gene Therapy

David W. Russell, MD, PhD
Collagen Gene Targeting and Osteogeneis Imperfecta

Scientific Symposium 312
8:00 am - 10:00 am
Immunology of Gene Therapy: Fundamental Immunology for Gene Therapy
Room: 314/315 – 316/317
Chair: Rafi Ahmed, PhD

Speakers:
Rafi Ahmed, PhD
Memory CD8 T Cell Differentiation During Viral Infection

Hao Shen, PhD
CD8+ T Cell Responses and Memory

Arlene H. Sharpe, MD, PhD
Roll of T Cell Costimulatory Pathways in Regulatory T Cell Activation and Tolerance

Scientific Symposium 313
8:00 am - 10:00 am
Respiratory Tract: Lung Cell Biology Update - Identifying Gene Therapy Targets and Improving Gene Transfer Efficiency
Room: 321/323
Chair: Larry G. Johnson, MD

Speakers:
John F. Engelhardt, PhD
Stem Cell Biology in the Adult Airways

Joseph Zabner, MD
Impact of Cell Polarity and Injury/Repair Responses on Gene Transfer Efficiency

David Erle, MD
Use of Genomics Approaches to Identify Targets for Gene Based Therapies in Asthma

Scientific Symposium 314
8:00 am - 10:00 am
Viral: New Viral Vector Systems
Room: 339/342
Chair: Brian P. Sorrentino, MD

Speakers:
Noriyuki Kasahara, MD PhD
New Strategies for the Therapeutic use of Tumor-Selective Replication-Competent Retrovirus Vectors: Teaching Old Viruses Some New Tricks

Joseph C. Glorioso, PhD
Novel Design and Application of HSV Vectors

Andrew Davidoff, MD
Self Complementary AAV for Hemophelia B Gene Therapy

Break
10:00 am - 10:15 am

Scientific Symposium 320
10:15 am - 12:15 pm
Embryonic/Somatic Stem Cells & Tissue Engineering: How to Define Stem and Progenitor Cells for Tissue Repair
Room: Ballroom
Chair: Darwin J. Prockop, MD, PhD

Speakers:
Evan Snyder, MD, PhD
Defining the Differentiation State of the Stem Cell Best Suited for CNS Therapy

Maureen A. Gannon, PhD
Temporal Regulation of the Pancreatic Transcription Factor HNF6 is Essential for Beta Cell Maturation

Lonnie D. Shea, PhD
Tissue Inductive Scaffolds as Controllable Cellular Microenvironments

Scientific Symposium 321
10:15 am - 12:15 pm
Hemopoietic: Gene Therapy for Genetic Diseases - Translational Models
Room: 314/315 – 316/317
Chair: Alice F. Tarantal, PhD

Speakers:
Donald B. Kohn, MD
Gene Therapy for Adenosine Deaminase-Deficient SCID

Michel Sadelain, MD PhD
Globin Gene Transfer for Beta-Thalassemia and Sickle Cell Disease: Strategies, Models, and Translation

Mark S. Sands, PhD
Gene Therapy for Lysosomal Storage Diseases

Scientific Symposium 322
10:15 am - 12:15 pm
Infectious Disease: Gene Therapy for Acute Infections
Room: 339/342
Chair: Geoff Symonds, PhD

Speakers:
Carl Merril
The Potential Phage Therapy for Acute Infections

David A. Suhy, PhD
Mechanistic Considerations for the Application of RNAi Against Infectious Viruses

Geoff Symonds, PhD
Ribozymes for HIV/AIDS

Scientific Symposium 323
10:15 am - 12:15 pm
Neural Disorders: Mechanisms & Experimental Therapies for Neurodegenerative Diseases
Room: 318/320
Chair: Xandra Breakefield, PhD

Speakers:
Pedro Gonzalez-Alegre, MD
Therapeutic RNA Interference for DYT1 Dystonia

Nick Mazarakis, PhD
ALS

Armin Blesch, PhD
Neurotrophin Gene Therapy for Alzheimer’s Disease

Scientific Symposium 324
10:15 am - 12:15 pm
Nonviral Gene Therapy: Targeting, Intracellular Trafficking and Regulation of Transgene Expression
Room: 327/329
Chair: Dexi Liu, PhD

Speakers:
Ernst Wagner, PhD
Synthetic Vectors for Tumor Targeting

Alan Verkman, MD, PhD
Intracellular Trafficking for Nonviral Gene Delivery

Mark A. Kay, MD PhD
Mechanisms Controlling Persistent Episomal Gene Expression

Break
2:30 pm – 2:45 pm

Simultaneous Oral Abstract Sessions
2:45 pm - 5:00 pm

Oral Abstract Session 340
RNA Virus Vectors I: Novel Vectors
Room: 327/329
Moderators: Donald B. Kohn, MD, and Karen E. Pollok, PhD
(Abstracts #367 - #375)

Oral Abstract Session 341
Adenovirus Vectors: Virus/Cell Interactions
Room: 314/315
Moderators: Andrea Amalfitano, DO, PhD, and Philip L. Leopold, PhD
(Abstracts # 376 - #384)

Oral Abstract Session 342
DNA Vectorology: Transposons and Chromatin Effects
Room: 345/346
Moderators: Michele P. Calos, PhD, and Perry B. Hackett, PhD
(Abstracts #385 - #393)

Oral Abstract Session 343
Chemical and Molecular Conjugates: Delivery Mechanisms
Room: 343-344
Moderators: Suzie H. Pun, PhD, and Hai-quan Mao, PhD
(Abstracts #394 - #402)

Oral Abstract Session 344
Metabolic Disorders & Lysosomal Storage Diseases
Room: 337/338
Moderators: Barry J. Byrne, MD, PhD, and Mark S. Sands, PhD
(Abstracts #403 - #411)

Oral Abstract Session 345
Neurologic
Room: 324/326
Moderators: Michael Kaplitt, MD, PhD and Nicole Déglon, PhD
(Abstracts #412 - #420)

Oral Abstract Session 346
Cancer-Immunotherapy: Signal Transduction and Antigen Presentation
Room: 316/317
Moderators: Katherine A. Whartenby, PhD, and Pedro Lowenstein, MD, PhD
(Abstracts #421 - #429)

Oral Abstract Session 347
Cancer-Targeted Gene Therapy: Oncolytic Viruses
Room: 339/342
Moderator: William F. Benedict, MD and Juan Fueyo, MD
(Abstracts #430 - #438)

Oral Abstract Session 348
Genetic Modification and Animal Models
Room: 321/323
Moderators: Brian P. Sorrentino, MD and Denise Sabatino, PhD
(Abstracts #439 - #447)

Oral Abstract Session 349
Stem Cell Therapy
Room: 318/320
Moderators: Ramesh Akkina, DVM, PhD, and Duncan J. Stewart, MD, FRCPC
(Abstracts #448 - #456)

Poster Session II
5:00 pm - 7:30 pm

RNA Virus Vectors I
(Abstracts 457 through 483)

AAV Vectors
(Abstracts 484 through 513)

DNA Vectorology
(Abstracts 514 through 540)

Physical Methods of Delivery
(Abstracts 541 through 558)

Muscle and Connective Tissue I
(Abstracts 559 through 579)

Infectious Diseases & Vaccines
(Abstracts 580 through 611)

Immuno-Gene Therapy Strategies II
(Abstracts 612 through 636)

Cancer-Targeted Gene Therapy: Adenovirus and Herpes Virus
(Abstracts 637 through 658)

Hematologic
(Abstracts 659 through 682)

Lung and Respiratory Disease
(Abstracts 683 through 702)

Oligonucleotide Therapies A
(Abstracts 703 through 722)

Satellite Symposium 350
7:00 pm - 10:00 pm
Vector Induced Tumors: Fact or Fiction?
Room: 339/342
Chair: James M. Wilson, MD, PhD

Speakers:
Mark S. Sands, PhD
Incidence of Hepatocellular Carcinoma in Normal and MPSUII Mice Following Intravenous AAV2 Injection

James M. Wilson, MD, PhD
Tumor Formation in Mice Following AAV to Liver

David W. Russell, MD, PhD
Genomic Rearrangements Associated with AAV Integration

Thomas J. Hope, PhD
Biology of WPRE Sequences

Michael Themis, PhD
Oncogenesis Following Delivery of Lentivirus Vectors to Fetal and Neonatal Mice

Susan M. Kingsman, PhD
Integration Characteristics and Safety Features of Lentiviral Vectors

• Wednesday, May 31
• Thursday, June 1
• Friday, June 2
• Saturday, June 3
• Sunday, June 4