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...
Main Authors: | , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Publishing Group
2017-03-01
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Series: | Scientific Reports |
Online Access: | https://doi.org/10.1038/s41598-017-00152-5 |