This federal Project Grant award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) provides $671,240 to the University of Washington to develop improved gene replacement and editing therapies for Duchenne muscular dystrophy (DMD).
The key objectives are to: (1) refine novel technologies for expressing full-length and mini-dystrophin proteins, using split inteins to enable delivery at higher functionality than current micro-dystrophin approaches; and (2) develop enhanced dystrophin gene editing methods to maximize expression and duration, with a focus on targeting satellite cells. This research leverages recently described AAV capsid variants to enable more potent gene therapies at lower vector doses. The 5-year project runs from January 2025 through December 2029.
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