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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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Belbellaa, Brahim, Reutenauer, Laurence, Messaddeq, Nadia, Monassier, Laurent, Puccio, Hélène
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
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Original Article
Online adgang: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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