Two New Pimelic Diphenylamide HDAC Inhibitors Induce Sustained Frataxin Upregulation in Cells from Friedreich's Ataxia Patients and in a Mouse Model

BACKGROUND: Friedreich's ataxia (FRDA), the most common recessive ataxia in Caucasians, is due to severely reduced levels of frataxin, a highly conserved protein, that result from a large GAA triplet repeat expansion within the first intron of the frataxin gene (FXN). Typical marks of heterochr...

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Bibliographic Details
Main Authors: Rai, Myriam, Soragni, Elisabetta, Chou, C. James, Barnes, Glenn, Jones, Steve, Rusche, James R., Gottesfeld, Joel M., Pandolfo, Massimo
Format: Artigo
Language:Inglês
Published: Public Library of Science 2010
Subjects:
Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC2809102/
https://ncbi.nlm.nih.gov/pubmed/20098685
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0008825
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