Lentiviral vectors can be used for full-length dystrophin gene therapy

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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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Detalles Bibliográficos
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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5356018/
https://www.ncbi.nlm.nih.gov/pubmed/28303972
http://dx.doi.org/10.1038/srep44775

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5356018/
https://www.ncbi.nlm.nih.gov/pubmed/28303972
http://dx.doi.org/10.1038/srep44775

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