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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Sci Rep
Päätekijät: 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
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Nature Publishing Group 2017
Aiheet:
Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC5356018/
https://ncbi.nlm.nih.gov/pubmed/28303972
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep44775
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