Excellence in Research Awards for Students and Postdoctoral Fellows Announced (top three student abstracts/top three postdoctoral fellow abstracts)

Workshop WS210
Education - How To Do an Academic Clinical Trial
Chair: Katherine High, MD

Speakers:

Greg Podsakoff, MD
Gene Therapy Trials at a Children's Hospital

Kenneth Cornetta, MD
Ex Vivo Transduction of Hematopoietic Cells

Katherine High, MD
Hybrid Vigor: Academia/Industry Collaboration in Gene Transfer for Hemophilia

Richard A. Knazek, MD
Vector Production and Toxicology Studies Available Through the National Gene Vector Laboratories

Philippe Bishop, MD
Long-Term Clinical Follow-up in Gene Therapy Studies

David W. Russell, MD, PhD
Gene Targeting with AAV Vectors

Peter Glazer, MD, PhD
Targeted Genome Modification Via Triple Helix Formation

George Dickson, PhD
Gene Conversion of Apolipoprotein-E Polymorphisms by In Situ Chimeraplasty

Olga Igoucheva, PhD
Targeted Gene Alteration by Oligonucleotides in Mammalian Cells

Kaarin K. Goncz, PhD
Sequence Modification of the Endogenous B-globin Gene in Human Hematopoietic Progenitor/Stem Cells by SFHR

Robert Kapsa, PhD
Targeted Gene Conversion in the Muscular Dystrophies

Workshop WS212
Musculo-Skeletal - How Can We Improve Bone and Cartilage Healing?
Chair: Paul D. Robbins, PhD

Speakers: 

Wim Van Den Berg, PhD
Local Gene Transfer of Growth Factors

Steven C. Ghivizzani, PhD
Direct Gene Delivery to Cartilage Defects

Dan Gazit, MD, PhD
Engineered Adult Mesenchymal Stem Cells (AMCs): A Platform for Gene Therapy and Skeletal Tissue Engineering

Axel Baltzer, MD
Enhancement of Bone Healing by Ad-BMP-2

Steven A. Goldstein, PhD
Localized Gene Therapy to Enhance Bone Repair

Workshop WS213
Neural Disorders I - Altering Vectors to Improve CNS Expression and/or Delivery
Chair: Pedro Lowenstein, MD, PhD

Speakers: 

Jacques Mallet, PhD
Lentiviral Vectors for CNS: Influence of Pseudotyping, Promotor and Post-Transcriptional Elements on Transgene Expression

Thomas Wickham, PhD
Developing Genetically Targeted Adenovirus Vectors for the CNS

Pedro Lowenstein, MD, PhD
Viral Vectors' Interactions with Innate and Adaptive Immunity

Nick Mazarakis, PhD
EIAV Lentiviral Vectors: Alternate Pseudotyping and Delivery to the CNS

David J. Fink, MD
HSV Vectors: Promoter Choice for CNS Applications

Workshop WS214
Non-Viral I - Potential Clinical Applications of Non-Viral Vectors
Co-Chairs:

Ronald K. Scheule, PhD
Sean M. Sullivan, PhD

Speakers: 

Nelson Yew, PhD
Correction of the x-galactosidase Deficiency in Fabry Mice Using Synthetic Gene Delivery

Carol Miao, PhD
Non-Viral Gene Therapy for Hemophilia

James G. Hecker, MD, PhD
Clinical Application: When is Short-term Expression Better?

Michael Coleman, PhD
Del-1 Gene Therapy for Ischemic Heart Disease

Workshop WS215
Respiratory Tract I - Asthma
Co-Chairs:

Phillip Factor, DO
Joel Kline, MD

Speakers: 

Manel Jordana, PhD
Immune Regulation of the Airway Microenvironment with Cytokine Gene Transfer

Art Krieg, MD
Immunotherapy with CpG Oligos

Joel Kline, MD
CpG Oligodeoxynucleotides and Their Role in the Prevention and Treatment of Asthma

Dale T. Umetsu, MD, PhD
Timl, A Novel Asthma Susceptibility Gene

Workshop WS216
Viral Vectors I - Use of Alternative Viral Serotypes for Virus Targeting or Evading the Immune System
Chair: Christopher Walsh, MD, PhD

Speakers: 

Barry J. Byrne, MD, PhD
Use of AAV-1 in a Model of Inherited Cardio Myopathy

Terence Flotte, MD
Targeting Recombinant Adeno-Associated Virus Vectors to Enhance Gene Therapy for Type I Diabetes

Paul B. McCray, Jr., MD
Pseudotyping FIV Vectors to Target Airway Epithelia

Joseph Zabner, MD
New Adeno Associated Virus for Gene Transfer to the Airways

Christine Halbert, PhD
Mouse Airway Transduction Following Recombination Between AAV Vectors Carrying Parts of a Larger Gene

Roland W. Herzog, PhD
Alternative AAV Serotypes for Treatment of Hemophilia B - Implications for Efficacy and Immuneresponses

Speakers: 

Philip Leopold, PhD
Endothelial Cells as Targets for Adenovirus-Mediated Gene Transfer

Kenneth Wu, MD, PhD
Gene Transfer Therapy with Vasoprotective Enzymes

Frank J. Giordano, MD
Transduction of the Myocardium via the Microvasculature

Geoff Akita, DVM, PhD
Modulation of Gene Expression in Cardiovascular Disease by HIF-12/UP16 Gene Transfer

Workshop WS218
Industrial Liaison - Product Licensure Issues
Co-Chairs: 

Parris Burd, PhD
Richard Gregory, PhD

Speakers: 

John Monahan, PhD
Licensure Issues for Gene Therapy Treatments for Hemophilia and Neurological Diseases

Thomas Reynolds, MD, PhD
Licensure Issues for CF Gene Therapy

Jeffrey Friedman, MD
Gene Therapy Product Licensure Issues

Jon Norman, PhD
Development of Quality Control Release Assays for DNA-Based Products

Douglas J. Jolly, PhD
Fun and Games in Biotechnology - What to Patent, What to License and What to Pay

Workshop WS220
Hemopoietic - Improving Stem Cell Transduction
Chair: Peter Quesenberry, MD

Speakers: 

Peter Quesenberry, MD
The New Stem Cell Biology

Hans-Peter Kiem, MD
Stem Cell Gene Transfer/Therapy: Recent Advances in Large Animal Models

Derek Persons, MD, PhD
In Vivo Selection and Gene Therapy for the Hemoglobin Disorders

Harry L. Malech, MD
Advances in Vectors for Correction of Chronic Granulomatous Disease

Fabio Candotti, MD
Hematopoietic Stem Cell Transduction as Treatment for Adenosine Deaminase Deficiency

Workshop WS221
Neural Disorders II - Methods to Improve Distribution of Vectors and Proteins to CNS
Chair: Beverly L. Davidson, PhD

Speakers:

Krys Bankiewicz, MD, PhD
Convection-Enhanced Delivery, Heparin, Axonal Transport and Neuroimaging of AAV-Mediated Gene Delivery to the Brain

Beverly L. Davidson, PhD
Taking Advantage of the PTD from HIV TAT

Mark S. Sands, PhD
Correction of Pathology in the Brain of Murine Model of Lysosomal Storage Disease Following Intraocular Gene Therapy

William M. Pardridge, MD
Non-Invasive, Non-Viral Gene Therapy to the Brain

Workshop WS222
Non-Viral II - Improvements in Non-Viral Vector Development
Co-Chairs:

Dexi Liu, PhD
Jon Wolff, MD

Speakers: 

Robert Debs, MD
Role of Vectors: Carrier Interaction in Determining Vector Efficiency

James E. Hagstrom, PhD
Intravascular Mediated Gene Delivery Using Non-Viral Vectors

Kevin G. Rice, PhD
Reductively Triggered Delivery of DNA Intracellularly

Ruxandra Draghia-Akli, MD, PhD
Ligand Regulated Plasmid DNA Vectors

David Putnam, PhD
Phagosomolytic Microparticulates for DNA Vaccine Delivery

Sandro Rusconi, PhD
Selective Nuclear Translocation of Steroid-Decorated Transgenes

Workshop WS223
Genetic Disease II - Clinical Trials in Hemophilia
Chair: Mark Kay, MD, PhD

Speakers: 

Robert E. Sobol, MD
Helper Dependent "Gutted" Adenoviral Factor VIII Vector for Hemophilia A: Phase I Clinical Trials and Future Directions

David A. Roth, MD
Non-Viral Ex Vivo Gene Therapy for Hemophilia A

Katherine High, MD
Safety Issues in AAV-Mediated Gene Transfer for Hemophilia

Workshop WS224
Viral Gene Transfer Vectors II - New Viral Vectors
Chair: Michele P. Calos, PhD

Speakers: 

James M. Wilson, MD, PhD
New AAVs from Nonhuman Primates

Paul B. McCray, Jr., MD
Optimizing FIV Vectors for Gene Therapy Applications

David W. Russell, MD, PhD
Foamy Virus Vectors

Stephen Yant, PhD
A Novel Adenovirus/Transposon Chimeric Vector for Stable Gene Transfer In Vivo

Michele P. Calos, PhD
Prolonged Expression Through Stable Extrachromosomal Vectors or Site-Specific Genomic Integration

Workshop WS225
Respiratory Tract II - Retargeting Vectors to the Pulmonary Vascular Endothelium
Chair: Paul N. Reynolds, MD, PhD

Speakers: 

Paul N. Reynolds, MD, PhD
Conjugate-based Adenoviral Retargeting

Andrew H. Baker, PhD
Development of Endothelial Cell-Selective Viral Vectors by Genetic Modification of Capsid Proteins

Raymond J. Pickles, PhD
Retargeting Adenovirus to 7-Transmembrane Receptors

Jeffrey S. Bartlett, PhD
Development of Targeted AAV-Based Vector Systems

David A. Sanders, PhD
Principles and Strategies of Targeting Gene Delivery

Catherine O'Riordan, PhD
Dressing Up Adenoviruses to Modify Their Tropism

Workshop WS226
Ethics - Can We Make Germ Line Modification an Acceptable Procedure?
Chair: Mark S. Frankel, PhD

Speakers: 

Mark S. Frankel, PhD
Modifying the Germ Line: Public Understanding, Approval and Oversight

Michael Blaese, MD
Germ Line Modification - Intended or Accidental

Jacques Cohen, PhD
Is Ooplasmic Transfer Germ Cell Modification?

Philip D. Noguchi, MD
FDA Perspectives

Gregory Stock, PhD
Germ Line Enhancement: How, When, and Why

Erik Parens, PhD
Should We Make Germ Line Modification an Acceptable Procedure?

Workshop WS227
Special - Herpes Virus Vectors for Gene Transfer to the Peripheral Nervous System In Vivo
Co-Chairs

Joseph Glorioso, PhD
David J. Fink, MD

Michel Pohl, PhD
Gene Therapy of Inflammatory Pain

Michael Chancellor, MD
HSV Gene Transfer to the Bladder and Penis

Xandra Breakefield, PhD
HSV Amplicon Vector - Mediated Gene Delivery to Spontaneous Schwannomas in Mouse Model of Neurofibromatosis Type 2

David J. Fink, MD
HSV-Mediated Gene Transfer in the Treatment of Pain

Joseph Glorioso, PhD
Treatment of Peripheral Neuropathy Using HSV Gene Vectors

Workshop WS228
Cancer - How to Design Cancer Gene Therapy Clinical Trials
Chair: Philip Felgner, PhD

Speakers: 

Adrian P. Gee, PhD
The Contribution of the GMP Facility to Cancer Gene Therapy Clinical Trials

Bambi Grilley, RPH, CCRA, CCRC, CIP
Regulatory Requirements for Gene Therapy Clinical Trials

James A. Merritt, MD
The Clinical Development Plan as a Framework for Designing Cancer Gene Therapy Studies

Claudia Mickelson, PhD
Dealing with "Novel" Issues in Clinical Gene Transfer Trials

Philippe Bishop, MD
FDA's Gene Therapy Patient Tracking System Initiative

Peter F. Thall, PhD
Innovative Statistical Designs for Gene Therapy Trials

Meet the Expert Lunch ME230
Hematopoietic Stem Cells
David Bodine, PhD

Corporate Symposium 250
HIV-Based Lentiviral Vectors in Human Gene Therapy
(Supported by VirXsys)
The goal of this program is to provide an overview on the potential use of HIV-based lentiviral vectors for human gene therapy. HIV-vectors have shown considerable promise as vectors for efficient and stable gene transfer into human cells. The speakers will provide an overview on vector design, production methods, examples of high transduction efficiency of human cells and potential disease target applications.

Session Chair/Moderator: Boro Dropulic, PhD

Speakers:

Inder Verma, PhD
The Biology of HIV-Based Lentiviral Vectors

Donald B. Kohn, MD
Stem Cell Gene Therapy for HIV/AIDS Using HIV-Vectors

Boro Dropulic, PhD
Pre-clinical Characterization of HIV-Vectors for HIV/AIDS Gene Therapy

Theodore Friedmann, MD 
The Biology of VSV-G Pseudotyped HIV-Vectors

Gabor Veres, PhD
TBA