Genetic Therapies for Genetic Diseases: Results and Lessons from Recent Successes
Air date: Wednesday, May 29, 2013, 3:00:00 PM
Description: Wednesday Afternoon Lecture Series
The disease burden represented by genetic disease is high. Genetic diseases account for nearly half of all admissions to children's hospitals, yet therapeutic options for many of these are limited. The goal of gene therapy is to harness the power of the Human Genome Project to provide better alternatives for those with genetic diseases. After two decades of clinical investigation of gene therapy, the field is able to point to several successes and one licensed product (an AAV vector for lipoprotein lipase deficiency). This lecture will review the vectors that have performed best in the setting of genetic disease and will focus on genetic diseases for which promising results have been reported using AAV vectors, including hemophilia B and Leber's congenital amaurosis, a rare form of inherited retinal degeneration. Delineation of major obstacles to clinical success will be reviewed and solutions discussed. Finally, in more recent work, it has been possible to combine genome editing approaches with AAV-mediated gene transfer to effect correction of mutations in situ. Strategies for using this approach to correct both plasma protein deficiencies and lysosomal storage disorders will be reviewed.
Author: Katherine A. High, M.D., Investigator, Howard Hughes Medical Institute William H. Bennett Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania Director, Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia
Runtime: 00:58:01
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