R R - American Society of Gene & Cell Therapy

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The American Society of Gene Therapy’s 2004 7th Annual Meeting final program Wednesday, June 2 Pre-Meeting Sessions Programs of Excellence in Gene Therapy 2:00 pm – 3:45 pm Room: 205 A-D Co-Chairs Sonia Skarlatos, PhD George Stamatoyannopoulos, MD Speakers Joseph C. Glorioso, PhD - The Pittsburgh PEGT Cardiovascular Disease Center Neil R. Hackett, PhD - Capsid Modified Adenoviral Vectors for Anti-Pseudomonas Vaccination Hans-Peter Kiem, MD - Stem Cell Gene Transfer and Selection: Implications for the Treatment of Genetic Diseases Katherine A. High, MD - Novel Therapeutic Approach for Hemophilia Using Gene Delivery of an Engineered Secreted Activated FVII Understanding and Planning Biodistribution and Toxicology Studies 2:00 pm – 3:45 pm Room: 211 A-D Chair Larry A. Couture, PhD Speakers Joy Cavagnaro, PhD, DABT, RAC - Considerations in Study Design: Optimizing Value Added for Communicating Risk John G. Page, PhD, DABT - Planning and Design of Preclinical Toxicology and Biodistribution Studies Theresa Chen, PhD - Preclinical Assessment of Gene Therapy Products: CBER Perspective Lorraine Matheson - Accessing and Contributing to Available Database Resources for Biodistribution/ Toxicology Studies Education Session I 4:00 pm – 5:30 pm ES100 - Retrovirus Vectors Room: 205 A-D Retroviral vectors created on the basis of simple gammaretroviruses such as murine leukemia viruses (MLV) represent a well characterized but still surprising tool for stable gene transfer. Efficiency and safety of these vectors depend on the multiplicity of insertions per host genome, on the pool size and specific targeting of the relevant cell type, and on the choice of appropriate cis-acting elements of the retroviral transgene. Detailed studies of retroviral vector elements are ongoing to improve the performance of current vectors, and to derive mechanisms that are of principal interest for the design of future recombinant vectors. Further progress in retroviral vector design will greatly rely on the correct choice and proper integration of non-MLV sequences in transgenes and particles, and may finally lead to the construction of ideal ferries for stable transgene insertion. Chair Christopher Baum, MD Speakers Christopher Baum, MD - Simple Retroviruses: Not So Simple Vectors Didier Trono, MD - Lentiviral Vectors for Basic Studies and Therapeutic Purposes Luigi Naldini, MD, PhD - Lentiviral Vectors ES101 - AAV Vectors Room: 102 A-C Adeno-associated virus (AAV) is emerging as a popular vector system for long term gene transfer. This workshop will present the basic biology of AAV and review its utility in gene therapy applications. The session will begin with talks that define the biology of the virus and the differences between some of the serotypes that are commonly used as vectors for gene transfer. Other talks will discuss the transduction pathway of the vectors in the cell, trafficking events that can limit vector transduction, and vector production and preparation. Chair Jay A. Chiorini, PhD Speakers Jay A. Chiorini, PhD - The ABCs of AAV Guangping Gao, PhD - Primate Derived AAVs as Efficient Gene Transfer Vectors: Virus Evolution and Vector Development Giovanni Di Pasquale, PhD - What AAV Needs to Express the Transgene: AAV/Host Cell Interactions Richard O. Snyder, PhD - Recombinant AAV Vector Manufacturing 29
The American Society of Gene Therapy’s 2004 7th Annual Meeting final program Wednesday, June 2 ES102 - Adenovirus Vectors Room: 102 D-F The goal of the Adenovirus Education Session is to survey the current state-of-the-art in our development and use of adenovirus gene transfer vectors. The session will begin with a discussion of the molecular biology of adenovirus including the production of replication –deficient vectors and packaging cell lines, the benefits and deficits of deletions of subsets or all the viral genes in the vector, the logic behind conditionally replicating adenoviruses, and variations relating to the Transgene expression cassette. The second topic of discussion will address the efficiency with which adenovirus interacts with target cells to deliver its genome to the nucleus. This discussion will include the basic infection pathway, altered vector tropism, variables relating to target cell type and physiology, and in vivo vector trafficking. The final topic of the session will be the interaction between adenovirus and the immune system including innate immunity, acquired immunity, and strategies for circumventing immune system barriers. Chair Philip L. Leopold, PhD Speakers Philip L. Leopold, PhD - Adenovirus Education Session James M. Wilson, MD, PhD - Innate and Required Immunity to Adenoviral Vectors Neil R. Hackett, PhD - Molecular Biology of Adenovirus Vectors ES103 - Non-Viral Vectors Room: 103 A-C In seeking to develop non-viral vector systems for nucleic delivery, researchers aim to replace viral vectors with physical techniques or chemistry-based self-assembly complexes as the preferred means for nucleic delivery. So far this aim has not yet been realized due to a number of significant problems that continue to frustrate research into the development of efficient non-viral vectors for nucleic acid delivery. However, these problems are approaching solution with a number of non-viral systems currently under development. Therefore, the overarching objective of this education session is to give a realistic impression of non-viral vector systems not only in terms of “where we are now” but “where we will be soon”. In this way the participant will be able to appreciate that non-viral vectors are not as far away from viral vectors as is commonly believed, and that with their inherent advantages there is every likelihood going forward that non-viral vectors can compete favorably with viral vectors for applications in clinical gene therapy. Chair Andrew D. Miller, PhD Speakers Andrew D. Miller, PhD - Gene Therapy Needs Robust Synthetic Non-Viral Platform Technologies Yuhong Xu, PhD - Gene Delivery by Physical Methods Ian MacLachlan, PhD - Special Considerations for the Development of Nucleic Acid Based Drugs ES104 - Immunology Room: 200 A-C In vivo delivery of genes for therapeutic purposes and vaccines induces immune responses directed both against the vector as well as the gene-product. The goal of this educational session is to provide an overview of the role of immune responses in strategies for development of vector systems for gene therapy and vaccines. The overview of the immune response will include innate immunity, antigen processing and presentation, lymphocyte activation, induction of effecter responses, and regulation of the immune responses. There will be a presentation and discussion on the use of gene transfer to induce tolerance to therapeutic or transplant antigens. Several exciting new approaches involving mechanisms of deletion, energy, or suppression will be discussed. In addition experiences of immunologic factors influencing efficacy, and safety in clinical trials with viral vectors will be reviewed. Chair Narendra Chirmule, PhD Speakers Narendra Chirmule, PhD - Immune Responses to Vectors and Transgenes for Gene Therapy and Vaccines Roland W. Herzog, PhD - Using Gene Transfer to Induce Immune Tolerance Ben-Gary Harvey, MD - Immunologic Factors in Clinical Trials with Viral Vectors 30
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