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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Detaylı Bibliyografya
Yayımlandı:Sci Rep
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group UK 2017
Konular:
Article
Online Erişim: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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