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
Autores principales: Belbellaa, Brahim, Reutenauer, Laurence, Messaddeq, Nadia, Monassier, Laurent, Puccio, Hélène
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7648087/
https://www.ncbi.nlm.nih.gov/pubmed/33209958
http://dx.doi.org/10.1016/j.omtm.2020.08.018

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7648087/
https://www.ncbi.nlm.nih.gov/pubmed/33209958
http://dx.doi.org/10.1016/j.omtm.2020.08.018

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