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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

25 Jul 2017

Abstract excerpt

Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to treat DMD. Here we show that locoregional and systemic delivery of a rAAV2/8 vector expressing a canine microdystrophin (cMD1) is effective in...

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PMID: 28742067
DOI: 10.1038/ncomms16105

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