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...

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Bibliographic Details
Main Authors: Chiranjeevi eSandi, Madhavi eSandi, Sara eAnjomani-Virmouni, Sahar eAl-Mahdawi, Mark Adrian Pook
Format: Article
Language:English
Published: Frontiers Media S.A. 2014-06-01
Series:Frontiers in Genetics
Subjects:
FXN
Online Access:http://journal.frontiersin.org/Journal/10.3389/fgene.2014.00165/full