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 en:Sci Rep
Autores principales: Counsell, John R., Asgarian, Zeinab, Meng, Jinhong, Ferrer, Veronica, Vink, Conrad A., Howe, Steven J., Waddington, Simon N., Thrasher, Adrian J., Muntoni, Francesco, Morgan, Jennifer E., Danos, Olivier
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group UK 2017
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Article
Acceso en línea: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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