Lentiviral vectors can be used for full-length dystrophin gene therapy
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 viral vectors. Lentiviral vectors can...
Paper Details
Title
Lentiviral vectors can be used for full-length dystrophin gene therapy
Published Date
Mar 6, 2017
Journal
Volume
7
Issue
1
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