Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.

Abstract excerpt

Duchenne muscular dystrophy (DMD) remains a major challenge in genetic medicine due to the difficulty of achieving durable, body-wide restoration of dystrophin in post-mitotic muscle tissues. Although current therapies-including exon skipping and micro-dystrophin gene replacement-have demonstrated clinical feasibility, their benefits are limited by incomplete efficacy, mutation specificity, and the need for...

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