Duchenne muscular dystrophy (DMD) is a progressive, degenerative muscle disease, typically caused by out-of-frame mutations in the DMD gene. Potential therapeutic strategies, such as exon skipping or clustered, regularly interspaced, short palindromic repeats (CRISPR) and -associated protein (Cas) 9, aim to restore the DMD reading frame to turn Duchenne into the milder, allelic disease, Becker muscular dystrophy. Currently, there are no dystrophic mouse models that allow for testing of such therapies in the context of the human DMD sequence in vivo.

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