An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models

Friedreich ataxia (FRDA) is a genetic disease due to increased repeats of the GAA trinucleotide in intron 1 of the frataxin gene. This mutation leads to a reduced expression of frataxin. We have produced an adeno-associated virus (AAV)9 coding for human frataxin (AAV9-hFXN). This AAV was delivered b...

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Detalles Bibliográficos
Autores principales: Gérard, Catherine, Xiao, Xiao, Filali, Mohammed, Coulombe, Zoé, Arsenault, Marie, Couet, Jacques, Li, Juan, Drolet, Marie-Claude, Chapdelaine, Pierre, Chikh, Amina, Tremblay, Jacques P
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362356/
https://www.ncbi.nlm.nih.gov/pubmed/26015982
http://dx.doi.org/10.1038/mtm.2014.44

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362356/
https://www.ncbi.nlm.nih.gov/pubmed/26015982
http://dx.doi.org/10.1038/mtm.2014.44

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