Article
Translating HDAC inhibitors in Friedreich’s ataxia
21 Jul 2016
Abstract excerpt
Introduction: Friedreich ataxia (FRDA) is an autosomal recessive neurodegenerative disease caused by expansion of a GAA·TTC triplet in the first intron of the FXN gene, encoding the essential mitochondrial protein frataxin. Repeat expansion results in transcriptional silencing through an epigenetic mechanism, resulting in significant decreases in frataxin protein in affected individuals. Areas covered: We review...
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