FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 49Program ScheduleOral Abstract Session 2302:00 pm - 4:00 pmRoom: Marriott Ballroom Salons 2 & 3AAV Vectors: Vector TraffickingCo-ChairsDavid V. Schaffer, PhD & Xiao Xiao, PhD(Abstracts 1-8; see page 86)Oral Abstract Session 2312:00 pm - 4:00 pmRoom: Maryland SuiteDNA Vectorology & Gene TargetingCo-ChairsPerry B. Hackett, PhD & Matthew H. Porteus, MD, PhD(Abstracts 9-16; see page 86)Oral Abstract Session 2322:00 pm - 4:00 pmRoom: Wilson ABCNeurologic & Ophthalmic Gene & Cell TherapyCo-ChairsMichael Kaplitt, MD, PhD & Deborah J. Watson, PhD(Abstracts 17-24; see page 87)Thursday, May 20 thOral Abstract Session 2332:00 pm - 4:00 pmRoom: Thurgood Marshall WCancer – Immunotherapy T Cell StudiesCo-ChairsChiara Bonini, MD & Dean A. Lee, MD, PhD(Abstracts 25-32; see pages 87-88)Oral Abstract Session 2342:00 pm - 4:00 pmRoom: Marriott Ballroom Salon 1Cancer-Oncolytic Viruses ICo-ChairsWinald Gerritsen, MD PhD & David H. Kirn, MD(Abstracts 33-40; see page 88)
50American Society of Gene & Cell TherapyProgram ScheduleOral Abstract Session 2352:00 pm - 4:00 pmRoom: Virginia SuiteImmunologic & Host Responses in Gene & Cell TherapyCo-ChairsEvanthia Galanis, MD, DSc & Maciej S. Lesniak, MD, FACS(Abstracts 41-48; see pages 88-89)Thursday, May 20 thOral Abstract Session 2362:00 pm - 4:00 pmRoom: Delaware SuiteOligonucleotide & RNAi Therapeutics ICo-ChairsDieter C. Gruenert, PhD & Faye A. Rogers, PhD(Abstracts 49-56; see page 89)Outstanding New Investigator Symposium 2404:30 pm - 6:15 pmRoom: Marriott Ballroom Salons 1, 2 & 3Outstanding New Investigator SymposiumChairJohn J. Rossi, PhDSpeakersAlessandra Biffi, MDRendering Hematopoietic Stem Cell Transplantation Efficacious for the Treatment of Lysosomal DisordersTransplantation of hematopoietic stem cells (HSC) from healthy donors has been employed in Lysosomal Storage Disorders (LSD) for delivering a functional hydrolaseto the affected tissues. However, likely due to the slow kinetics of infiltration of the nervous system, the efficacy of the transplant is poor in LSD with severe neurologicmanifestations. The limited efficacy of the allogeneic procedure may be implemented by: i) understanding the mechanisms of myeloid infiltration into affectedtissues, and in particular into the brain, in order to favour and accelerate this phenomenon; ii) rendering the transplanted HSC and or their progeny more effectivethan normal donors’ cells in enzyme delivery by means of gene transfer to enhance the metabolic correction of the affected tissue. We have addressed these issuesattempting to develop an effective and safe therapeutic approach for different LSD with CNS involvement, for one of which we recently started clinical testing.EXHIBITOR PROSPECTUSfinal program
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FINAL PROGRAM - American Society of Gene & Cell Therapy

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