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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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書目詳細資料
發表在: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
格式: Artigo
語言:Inglês
出版: Nature Publishing Group UK 2017
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在線閱讀: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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