Lentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts

Friedreich ataxia (FRDA) is a progressive neurodegenerative disease caused by deficiency of frataxin protein, with the primary sites of pathology being the large sensory neurons of the dorsal root ganglia and the cerebellum. FRDA is also often accompanied by severe cardiomyopathy and diabetes mellit...

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Detalles Bibliográficos
Autores principales: Khonsari, H, Schneider, M, Al-Mahdawi, S, Chianea, Y G, Themis, M, Parris, C, Pook, M A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5143368/
https://www.ncbi.nlm.nih.gov/pubmed/27518705
http://dx.doi.org/10.1038/gt.2016.61

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5143368/
https://www.ncbi.nlm.nih.gov/pubmed/27518705
http://dx.doi.org/10.1038/gt.2016.61

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