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Increasing Frataxin Gene Expression with Histone Deacetylase Inhibitors as a Therapeutic Approach for Friedreich's Ataxia
The genetic defect in Friedreich's ataxia (FRDA) is the expansion of a GAA·TCC triplet in the first intron of the FXN ene, which encodes the mitochondrial protein frataxin. Previous studies have established that the repeats reduce transcription of this essential gene, with a concomitant decreas...
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| Hauptverfasser: | , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2013
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3766837/ https://ncbi.nlm.nih.gov/pubmed/23859350 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/jnc.12302 |
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