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Molecular Mechanisms and Therapeutics for the GAA·TTC Expansion Disease Friedreich Ataxia
Friedreich ataxia (FRDA), the most common inherited ataxia, is caused by transcriptional silencing of the nuclear FXN gene, encoding the essential mitochondrial protein frataxin. Currently, there is no approved therapy for this fatal disorder. Gene silencing in FRDA is due to hyperexpansion of the t...
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| Publicado no: | Neurotherapeutics |
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| Autor principal: | |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Springer International Publishing
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6985418/ https://ncbi.nlm.nih.gov/pubmed/31317428 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s13311-019-00764-x |
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