Article

Assessment of Therapeutic Potential of A Dual Aav Approach for Duchenne Muscular Dystrophy

2023-06-29

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a yet incurable rare genetic disease that affects the skeletal and cardiac muscles, leading to progressive muscle wasting and premature death. DMD is caused by the lack of dystrophin, a muscle protein essential for the biochemical support and integrity of muscle fibers. Gene replacement strategies for Duchenne muscular dystrophy (DMD) employing the adeno-associated virus (AAV)...

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Literature Corpus work: 7e6f1a70-e1a6-5f99-8904-51469872d82c
DOI: 10.20944/preprints202306.2000.v1

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