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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...
保存先:
| 出版年: | Neurotherapeutics |
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| 第一著者: | |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Springer International Publishing
2019
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| 主題: | |
| オンライン･アクセス: | 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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