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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Pubblicato in:Sci Rep
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group UK 2017
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Article
Accesso online: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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