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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Sandi, Chiranjeevi, Sandi, Madhavi, Anjomani Virmouni, Sara, Al-Mahdawi, Sahar, Pook, Mark A.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Frontiers Media S.A. 2014
Pynciau:
Genetics
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4042889/
https://ncbi.nlm.nih.gov/pubmed/24917884
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fgene.2014.00165
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