High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models

Friedreich ataxia (FA) is currently an incurable inherited mitochondrial disease caused by reduced levels of frataxin (FXN). Cardiac dysfunction is the main cause of premature death in FA. Adeno-associated virus (AAV)-mediated gene therapy constitutes a promising approach for FA, as demonstrated in...

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Detalles Bibliográficos
Publicado en:Mol Ther Methods Clin Dev
Autores principales: Belbellaa, Brahim, Reutenauer, Laurence, Messaddeq, Nadia, Monassier, Laurent, Puccio, Hélène
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7648087/
https://ncbi.nlm.nih.gov/pubmed/33209958
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.08.018
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