COMPOSITIONS AND METHODS FOR TREATING A MUSCULAR DYSTROPHY
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United States of America
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Jan 16, 2025
app pub date -
Aug 10, 2022
filing date -
Aug 10, 2022
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Abstract
The present disclosure provides novel synthetic nucleic acids and recombinant adeno-associated virus (rAAV) comprising the same, as well as methods of their use in the treatment of muscular dystrophies associated with a dystrophin mutation. Also provided are pharmaceutical compositions comprising a novel synthetic nucleic acid or rAAV of the invention, and a pharmaceutically acceptable carrier or excipient. Pharmaceutical compositions comprising an rAAV of the invention may be useful in gene therapy for the treatment of dystrophin-associated muscular dystrophies, such as Duchenne muscular dystrophy (DVD). Becker muscular dystrophy (BMD), and X-linked cardiomyopathy.
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