Article

<i>In vivo</i> base editing via single myotrophic adeno-associated viruses in dystrophic mouse muscle and satellite cells

2026-05-10

Abstract excerpt

Duchenne muscular dystrophy (DMD) is the most common, lethal X-linked neuromuscular disorder of childhood and is caused by mutations in the Dmd gene that disrupt dystrophin expression. Although adeno-associated virus-mediated gene therapies hold tremendous promise for DMD treatment, their clinical applications have been limited by dose-dependent vector and genome-level toxicities. Here, we developed and tested a...

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Literature Corpus work: 42d29134-99af-534a-a1eb-b0caab73a397
DOI: 10.64898/2026.05.09.721064

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