Article

Potential limitations of micro-dystrophin gene therapy for Duchenne muscular dystrophy

2022-10-05

Abstract excerpt

<h4>ABSTRACT</h4> Adeno-associated viruses (AAVs) expressing versions of truncated dystrophin (micro-dystrophins) are being delivered at high doses to patients with Duchenne muscular dystrophy (DMD) in clinical trials. We examined this strategy with two different micro-dystrophins, similar to those currently in clinical trials, in a severe mouse model of DMD, the D2. mdx mouse, using doses of AAV comparable to t...

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Literature Corpus work: 7f8dc4fb-9462-5827-a307-7991a7622e05
DOI: 10.1101/2022.10.02.510519

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