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<i>Large1</i> Gene Transfer in Older <i>myd</i> Mice with Severe Muscular Dystrophy Restores Muscle Function and Greatly Improves Survival

2021-10-28

Abstract excerpt

<h4>ABSTRACT</h4> Muscular dystrophy is a progressive and ultimately lethal neuromuscular disease due to lack of therapeutic options that restore muscle function. Gene editing and gene transfer hold great promise as therapies for various neuromuscular diseases when administered prior to the onset of severe clinical symptoms. However, the efficacy of these strategies for restoring neuromuscular function and improv...

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Literature Corpus work
9f9abc86-6d1f-53e2-9b17-dc16ab206f42
DOI
10.1101/2021.10.28.466309
Open publication

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<i>Large1</i> Gene Transfer in Older <i>myd</i> Mice with Severe Muscular Dystrophy Restores Muscle Function and Greatly Improves SurvivalDOI 10.1101/2021.10.28.466309
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