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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Detalles Bibliográficos
Autores principales: Sandi, Chiranjeevi, Sandi, Madhavi, Anjomani Virmouni, Sara, Al-Mahdawi, Sahar, Pook, Mark A.
Formato: Artigo
Lenguaje:Inglês
Publicado: Frontiers Media S.A. 2014
Materias:
Genetics
Acceso en línea: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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