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Our research is focused on elucidating key mechanisms responsible for pathologies observed in genetic muscle disorders (primarily in regards to the muscular dystrophies), and on the development of potentially permanent gene therapy approaches using CRISPR/Cas-based technologies. To this end, we have developed several muscle-specific in vivo gene-editing approaches utilizing adeno-associated viral (AAV) vectors to deliver CRISPR/Cas9 to striated muscles, and demonstrated their usefulness for correcting dystrophin expression and ameliorating pathophysiology in Duchenne muscular dystrophy. Current research efforts include an expanded use of applicable genome targeting technologies towards correction of genetic mutations in both post-mitotic myonuclei and in muscle stem cells, as well as on improving both the overall efficiency and safety of these methods. Several innovative in vitro & in vivo platforms are currently being designed to enable both rapid initial proof-of-principle testing of novel genome-corrective methods, and to facilitate functional validation of promising approaches prior to further development towards clinical application.