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Lentiviral vectors can be used for full-length dystrophin gene therapy
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 res...
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| Udgivet i: | Sci Rep |
|---|---|
| Main Authors: | , , , , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Nature Publishing Group UK
2017
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5427806/ https://ncbi.nlm.nih.gov/pubmed/28250438 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-017-00152-5 |
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