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Maximizing dystrophin functionality

Gene therapy for Duchenne muscular dystrophy is an extremely promising approach to therapy as it directly addresses the cause of the disorder- a lack of functional dystrophin protein. Our group has spent more than 30 year studying the structure and function of dystrophin and developing approaches for gene therapy. This work includes development of numerous micro-dystrophin clones, several of which are being tested in clinical trials. We also showed that AAV vectors could be used for systemic delivery of new genes to muscles, bodywide. However, while current AAV-microdystrophin vectors are showing significant benefit in the clinic, several limitations have been identified. Among these are a lack of full function from micro-dystrophins, lower levels of dystrophin production than are ideal, and some vector dose limitations due to adverse events. Our proposal explores a new way to make larger mini-dystrophins and even full-sized dystrophin by co-delivery of current and novel AAV vectors. Here we explore the potential of this system to improve upon approach for gene therapy of DMD and other neuromuscular disorders.
https://doi.org/10.55762/pc.gr.158724
Grantee: Jeffrey Chamberlain, PhD
Grant type: Restricted Research Grant
Award total: $233,243
Institution: University of Washington
Country: Washington, United States