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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...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Hindawi Publishing Corporation
2013
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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 |