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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Detaylı Bibliyografya
Yayımlandı:Mol Ther Methods Clin Dev
Asıl Yazarlar: Gérard, Catherine, Xiao, Xiao, Filali, Mohammed, Coulombe, Zoé, Arsenault, Marie, Couet, Jacques, Li, Juan, Drolet, Marie-Claude, Chapdelaine, Pierre, Chikh, Amina, Tremblay, Jacques P
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2014
Konular:
Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC4362356/
https://ncbi.nlm.nih.gov/pubmed/26015982
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2014.44
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