American Society of Human Genetics 2013 Annual Meeting

A Renaissance in Gene Therapy: New Tools and Clinical Trials

Moderators: Stephen Kaler, NICHD/NIH & Luk H. Vandenberghe, Harvard Med. School.

This session will focus on expanding knowledge and experience in the field of therapeutic gene transfer, with a focus on monogenetic disorders. Advances in somatic gene transfer have led to a renaissance in the field, in large part due to recent success in early clinical trials for hemophilia and inherited blinding diseases. This session will provide an update on recent improvements in the development of adeno-associated viral and lentiviral vector systems. Speakers will discuss preclinical and clinical advances that these novel technologies have enabled. Emerging data on the clinical application of viral gene therapy, as well as the contribution of this approach to understanding of disease mechanism and pathophysiology will be highlighted.

8:00am:   Adeno-associated viral vectors for gene therapy: Virus, vector, nanoparticle. L. H. Vandenberghe. Harvard Med. Sch.

8:30am:   Gene therapy for metabolic CNS disorders. R. G. Crystal. Weill Cornell Med. Col.

9:00am:   Brain-directed AAV gene therapy. S. Kaler. NICHD/NIH.

9:30am:   Lentiviral-mediated gene therapy for human disease: Extensive genetic engineering of hematopoiesis with therapeutic benefit in metachromatic leukodystrophy patients after lentiviral hematopoietic stem cell gene therapy. L. Naldini. San Raffaele Telethon Inst. for Gene Therapy, Milan.

Date & Time: Wednesday, October 23, 8am-10am

Location: Boston Convention & Exhibition Center, Room 253, Level 2, Convention Center

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