Epigenetic-based therapies for Friedreich ataxia
Friedreich ataxia (FRDA) is a lethal autosomal recessive neurodegenerative disorder caused primarily by a homozygous GAA repeat expansion mutation within the first intron of the FXN gene, leading to inhibition of FXN transcription and thus reduced frataxin protein expression. Recent studies have sho...
Main Authors: | , , , , |
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Format: | Article |
Language: | English |
Published: |
Frontiers Media S.A.
2014-06-01
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Series: | Frontiers in Genetics |
Subjects: | |
Online Access: | http://journal.frontiersin.org/Journal/10.3389/fgene.2014.00165/full |