Article

Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

2025-12-25

Abstract excerpt

Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), a progressive muscle wasting disorder caused by pathogenic variants in the DMD gene that typically disrupt the translation reading frame. This approach aims to modulate DMD pre-mRNA splicing to re-frame the transcript and generate an internally deleted but partially functional quasi-dystrophin protein. Four exon skipping drugs...

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Literature Corpus work: 67228791-1e3d-5d35-a0f1-9a2f96404bc9
DOI: 10.64898/2025.12.23.696144

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