AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects

Abstract excerpt

Duchenne muscular dystrophy (DMD) is caused by pathogenic sequence variants occurring in the DMD gene which lead to the loss of the dystrophin protein, a molecular 'shock absorber' that protects muscle from contraction-induced injury. The large size of the dystrophin open reading frame precludes delivery of the full-length protein using a single adeno-associated virus (AAV) vector, which led to the development of...

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