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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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Main Authors: Gottesfeld, Joel M., Rusche, James R., Pandolfo, Massimo
格式: Artigo
語言:Inglês
出版: 2013
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在線閱讀: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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