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Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich’s Ataxia
Friedreich’s ataxia (FRDA) is an inherited, multisystemic disorder predominantly caused by GAA hyper expansion in intron 1 of frataxin (FXN) gene. This expansion mutation transcriptionally represses FXN, a mitochondrial protein that is required for iron metabolism and mitochondrial homeostasis, lead...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Frontiers Media S.A.
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9157421/ https://www.ncbi.nlm.nih.gov/pubmed/35663795 http://dx.doi.org/10.3389/fgeed.2022.903139 |