Pharmacological Screening Using an FXN-EGFP Cellular Genomic Reporter Assay for the Therapy of Friedreich Ataxia

Friedreich ataxia (FRDA) is an autosomal recessive disorder characterized by neurodegeneration and cardiomyopathy. The presence of a GAA trinucleotide repeat expansion in the first intron of the FXN gene results in the inhibition of gene expression and an insufficiency of the mitochondrial protein f...

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Detalhes bibliográficos
Main Authors: Li, Lingli, Voullaire, Lucille, Sandi, Chiranjeevi, Pook, Mark A., Ioannou, Panos A., Delatycki, Martin B., Sarsero, Joseph P.
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2013
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3572186/
https://ncbi.nlm.nih.gov/pubmed/23418481
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0055940
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