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Epigenetics in Friedreich's Ataxia: Challenges and Opportunities for Therapy

Friedreich's ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by homozygous expansion of a GAA·TTC trinucleotide repeat within the first intron of the FXN gene, leading to reduced FXN transcription and decreased levels of frataxin protein. Recent advances in FRDA resear...

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Detalles Bibliográficos
Autores principales: Sandi, Chiranjeevi, Al-Mahdawi, Sahar, Pook, Mark A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Hindawi Publishing Corporation 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3590757/
https://www.ncbi.nlm.nih.gov/pubmed/23533785
http://dx.doi.org/10.1155/2013/852080