Article

Lentiviral vectors can be used for full-length dystrophin gene therapy

2017-03-17

Abstract excerpt

Abstract Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystrophin expression in patient muscle fibres. Current DMD gene therapy strategies rely on the expression of internally deleted forms of dystrophin, missing important functional domains. Viral gene transfer of full-length dystrophin could restore wild-type functionality, although this approach is restricted by the limited capacity of recombinant vi...

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Literature Corpus work: 8febf444-a91f-5010-ac5c-5d9ee9873eba
DOI: 10.1038/srep44775

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