Article

Dual S100A1 and ARC gene therapy as a treatment for DMD cardiomyopathy

2025-08-23

Abstract excerpt

<h4>ABSTRACT</h4> Duchenne muscular dystrophy (DMD) is a lethal pediatric striated muscle disease caused by loss of dystrophin for which there is no cure. Cardiomyopathy is the leading cause of death amongst individuals with DMD, and effective therapeutics to treat DMD cardiomyopathy are a major unmet clinical need. This work investigated adeno-associated viral (AAV) gene therapy approaches to treat DMD cardiomyo...

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Literature Corpus work: 3659d485-a49d-5e1f-96ed-4f8cd7e51089
DOI: 10.1101/2025.08.23.671924

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