FINAL PROGR AM - American Society of Gene & Cell Therapy

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AMERICAN SOCIETY OF GENE THERAPY • Final Program 27 SCIENTIFIC PROGRAM WEDNESDAY, JUNE 1, 2005 Education Session 112 Regulation of Transgene Expression The ability to regulate the timing and level of transgene expression opened new avenues in basic research applications and might become prerequisites in future clinical settings. Several gene regulation systems have been developed and successfully incorporated into viral and non-viral gene delivery systems. The session will begin with a general overview on the characteristics common to the various gene expression regulating systems. The following parts of the session will focus on the most common gene regulatory systems including the tetracycline, ecdysone, and the dimerizer systems. The last part of this session will cover new developments in regulating siRNA expression cassettes. Room: 265/266 Chair: Tal Kafri, PhD Speakers: Tal Kafri, PhD - General overview on transgene regulation with special emphasis on the tetracycline gene regulatory system Francesco Galimi, MD, PhD - Regulation of Transgene Expression: Vector Designs and Applications Jakob Reiser, PhD - Knockdown Control: Practical Approaches to Regulating shRNA Expression Education Session 113 NIH Grantsmanship & Funding Opportunities The goal of this session is to acquaint participants with training and career development application mechanisms and the review process at the NIH. Staff will give advice on what contributes a successful grant application and how to pick the right mechanism depending on the applicant’s expertise and education. There will be an opportunity for questions during the presentations and NIH staff will be available for individual questions. This session is intended for new investigators but should be informative for any investigator planning to submit an NIH grant application. Room: 120/124 Chair: Sonia I. Skarlatos, PhD Speakers: Sonia I. Skarlatos, PhD - Research Training and Career Development Programs Steven J. Zullo, PhD - Grant Review in the CSR Arun Srivastava, PhD - Writing and Reviewing NIH Grants: A Personal Odyssey Education Session 114 Gene Transfer to Muscle This educational session will focus on applications, methods and strategies for gene transfer to muscle tissue. The first lecture will give an overview of viral vectors for muscle gene transfer in vivo. The advantages and disadvantages of a variety of commonly used vectors with examples of their application will be discussed. The second lecture will focus on non-viral methods for gene transfer to muscle, which is uniquely suited for uptake of non-viral vectors. The final topic will be on ex vivo methods for gene transfer and appropriate vectors for transducing stem cells. Room: 123/127 Chair: Jeffrey Chamberlain, PhD Speakers: Jeffrey Chamberlain, PhD - Viral Gene Transfer Jon Wolff, MD - Non-Viral Gene Transfer Giuliana Ferrari, PhD - Cell Therapy for Muscular Dystrophy Education Session 115 Building a Strategy for Translational Medicine: Focus on Immunodeficiencies The goal of this session is to highlight scientific, technological and logistical aspects that bear on the successful translation and clinical implementation of gene therapy research. This year’s session will focus on the severe combined immune deficiencies (SCID). The faculty will discuss their experience with adenosine deaminase (ADA) deficiency, X-linked SCID and Wiskott-Aldrich syndrome, with emphasis on the strategic choices they have made and the hurdles they have confronted on issues such as vector/ promoter selection, small vs. large animal models, vector production, infrastructure development, protocol approval, patient selection and recruitment, quality assurance and regulatory oversight. Room: 230/231 Chair: Michel Sadelain, MD PhD Speakers: Donald B. Kohn, MD - ADA-Deficient SCID: Pathogenis and Treatments Marina Cavazzana-Calvo, MD, PhD - Severe Combined Immunodeficiency: Disease Models for New Therapetical Approaches Arthur Nienhuis, MD - Development of Gene Therapy for Wiskott-Aldrich Syndrome DINNER BREAK – 6:45 PM - 8:30 PM
28 8th Annual Meeting • June 1 - 5, 2005 • St. Louis SCIENTIFIC PROGRAM WEDNESDAY, JUNE 1, 2005 EDUCATION SESSION III – 8:30 PM - 10:00 PM Education Session 120 Novel Vector Systems Novel adenovirus and adeno-associated virus vectors have been developed recently, which enable the delivery of transgenes into target cells and tissues with efficiency and specificity. Application of these novel vectors in the delivery of therapeutic genes to treat inherited disorders as well as acquired diseases like cancer will be discussed. Room: 260/264 Chair: Savio L.C. Woo, PhD Speakers: Andre Lieber, MD, PhD - Gene Transfer Vectors Based on B-group Adenoviruses David T. Curiel, MD, PhD - Targetable/ Injectable Adenoviral Vectors for Cell-specific Gene Transfer In Vivo Guangping Gao, PhD - Efficient Gene Transfer Vectors Based on Novel Primate AAVs Education Session 121 AAV Vectors The goal of the AAV Education Session is to provide the community not only with a general understanding of this popular vector system, but to integrate this into a broader awareness of expectations when using these vectors in vitro and in vivo. Three topics will be discussed in this session. The first speaker will begin with a general background of AAV biology including a discussion of the molecular fate of recombinant genomes in vitro and in vivo. The second topic will concentrate on structural differences between AAV serotypes and how those differences can be utilized to improve the vector delivery. The final topic will focus on applications of AAV serotype delivery to specific target organs including performance comparisons between different serotypes. Room: 263/267 Chair: Joseph Rabinowitz, PhD Speakers: Joseph Rabinowitz, PhD - Exploring the Structure and Function of the AAV Virion Xiao Xiao, PhD - Recent Progress in AAV Vector Development K. Reed Clark, PhD - Wild-type and Recombinant AAV Biology - An Update Education Session 122 RNAi RNA interference refers to a cells natural ability to silence gene expression using short interfering RNAs (siRNAs). Inhibitory RNAs are derived from larger precursors transcribed from the genome, or introduced into cells artificially. The power of RNAi has recently been harnessed by the bench scientist to selectively inhibit the expression of diverse gene products to ascertain biological function, or as therapeutic tools for silencing pathogenic protein expression. This session will provide an overview of the protein complexes involved in RNAi, and will also illustrate various methods to accomplish RNAi in cells, tissues and animals. Room: 240 Complex Chair: Beverly L. Davidson, PhD Speakers: Beverly L. Davidson, PhD - RNA Interference John J. Rossi, PhD - The Long and Short of RNAi Michael McManus, PhD - Developmental and Post-developmental Phenotypes of MicroRNA Ablation in the Mouse Education Session 123 Gene Transfer to the Central Nervous System In this session the three speakers will address specific issues pertaining to gene transfer in the nervous system. The broad goals are to convey to the audience the anatomic, immunologic and development aspects of the nervous system that are unique as a target for gene therapy. Dr. Fink will highlight the peripheral nervous system development, anatomy, function and disease related dysfunctions. Dr. Maria Castro will address neural immune interactions, locus of antigen presentation and consequences for gene transfer. Dr. Federoff will touch on special problems encountered in the translation of preclinical gene therapy. He will also briefly highlight issues related to the development of in utero CNS gene therapy. Room: 230/231 Chair: Howard J. Federoff, MD, PhD Speakers: Howard J. Federoff, MD, PhD - Translational Considerations for CNS Gene Therapy David J. Fink, MD - Gene Transfer to DRG for Treatment of Neuropathy or Chronic Pain Maria Castro, PhD - Regulated, Long Term Therapeutic Gene Expression for Neurological Gene Therapy
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156 NOTES 8th Annual Meeting • Ju
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