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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Bibliografske podrobnosti
izdano v:Sci Rep
Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group UK 2017
Teme:
Article
Online dostop: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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