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: | Online |
| Language: | English |
| Published: |
Frontiers Media S.A.
2014
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4042889/ |