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

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...

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Bibliographic Details
Main Authors: John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos
Format: Article
Language:English
Published: Nature Publishing Group 2017-03-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-017-00152-5

Internet

https://doi.org/10.1038/s41598-017-00152-5

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