Project 1
Gene Therapy

Principal Investigator: Jeffrey S. Chamberlain, Ph.D.
Co Investigators: Stephen Hauschka, Ph.D., Martin Childers, Ph.D., Joel Chamberlain, Ph.D., Guy Odom, Ph.D. and Zejing Wang, Ph.D.

This project is a collaborative effort among a diverse and highly experienced group of scientists with extensive expertise in the study of muscle biology, muscular dystrophy, gene expression, RNAi and gene therapy. We are working to adapt methods for AAV-mediated gene transfer to large animal models for DMD by focusing on expression cassette design, AAV serotype comparisons, delivery methods and scale-up to test the hypothesis that AAV vectors can be used to target all the major muscle groups of the body. In parallel, we will adapt AAV systems for targeting and studying the dominant disorder FSHD, using cell lines and mouse models. Together these studies could lead to human clinical trials for DMD using regional limb, diaphragm and cardiac delivery of AAV vectors, and will bring gene therapy for dominant disorders closer to pre-clinical testing. One clinical trial involving IM injection of AAV for DMD is already planned at Seattle Children’s Hospital, and together with the clinical trials infrastructure development in Project 2 these studies could lead to increased clinical interventions for both dominantly and recessively inherited muscular dystrophies.

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