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This review article provides an in-depth overview of gene therapy modalities — including gene replacement, gene editing, and gene inactivation — and how these st
New research has uncovered how rare CHKB variants disrupt RNA splicing, directly contributing to the pathology of megaconial congenital muscular dystrophy (MDCMC). T
The 2024 Muscular Dystrophy Association Summit brought together international experts to discuss the scientific, operational, and ethical challenges of bringing gene
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