PRODUCTION OF RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY
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United States of America
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Jan 16, 2025
app pub date -
May 13, 2022
filing date -
May 13, 2022
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Abstract
The present disclosure provides gene therapy vectors, such as recombinant adeno-associated virus (rAAV), produced in mammalian adherent cells cultured in suspension conditions, for expressing a human micro-dystrophin gene. The present disclosure also provides compositions and methods of using these rAAV to treat muscular dystrophy, such as, e.g., Duchenne Muscular Dystrophy.
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