COMBINATION THERAPY FOR TREATING MUSCULAR DYSTROPHY
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United States of America
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Feb 3, 2022
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Dec 11, 2019
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Dec 11, 2019
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Abstract
The invention described herein provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, that co-express a functional protein (such as a miniaturized human micro-dystrophin gene product) and one or more additional coding sequences for an RNAi sequence (siRNA, shRNA, miRNA), an antisense sequence, a guide sequence for a gene editing enzyme (such as an sgRNA for CRISPR/Cas9, or a crRNA for CRISPR/Casl2a), and/or a micro RNA, and methods of using the vectors to treat subjects suffering from a muscular dystrophy such as DMD/BMD.
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