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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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| Hauptverfasser: | , , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Frontiers Media S.A.
2014
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| Schlagworte: | |
| Online Zugang: | 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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