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Frataxin levels in peripheral tissue in Friedreich ataxia
Journal article   Open access   Peer reviewed

Frataxin levels in peripheral tissue in Friedreich ataxia

Michael Lazaropoulos, Yina Dong, Elisia Clark, Nathaniel R Greeley, Lauren A Seyer, Karlla W Brigatti, Carlton Christie, Susan L Perlman, George R Wilmot, Christoper M Gomez, …
Annals of clinical and translational neurology, Vol.2(8), pp.831-842
08/2015
DOI: 10.1002/acn3.225
PMCID: PMC4554444
PMID: 26339677
url
https://doi.org/10.1002/acn3.225View
Published (Version of record) Open Access

Abstract

Friedreich ataxia (FRDA) is an autosomal recessive ataxia resulting from mutations in the frataxin gene (FXN). Such mutations, usually expanded guanine-adenine-adenine (GAA) repeats, give rise to decreased levels of frataxin protein in both affected and unaffected tissues. The goal was to understand the relationship of frataxin levels in peripheral tissues to disease status.

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