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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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| Publicado no: | Sci Rep |
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| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5356018/ https://ncbi.nlm.nih.gov/pubmed/28303972 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep44775 |
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