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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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| Veröffentlicht in: | Mol Ther Methods Clin Dev |
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| Hauptverfasser: | , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
American Society of Gene & Cell Therapy
2020
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| Schlagworte: | |
| Online Zugang: | 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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