Article

Targeting lysosomal damage is a new therapeutic perspective for Duchenne Muscular Dystrophy

2025-02-13

Abstract excerpt

Duchenne Muscular Dystrophy (DMD), a muscle degenerative disease affecting young boys, arises from the loss of dystrophin. Current gene therapy approaches aim to restore a shortened form of dystrophin (microdystrophin) via Adeno-Associated Vector (AAV) delivery, but clinical studies show limited efficacy, emphasizing the need for improved strategies such as combined therapies. In this study, we identified lysosoma...

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Literature Corpus work: 9eecabd4-2b7e-5cf2-9a8d-5beabd018a4c
DOI: 10.1101/2025.02.11.637610

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