Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's ataxia

Inherited deficiency in the mitochondrial protein frataxin (FXN) causes the rare disease Friedreich's ataxia (FA), for which there is no successful treatment. We identified a redox deficiency in FA cells and used this to model the disease. We screened a 1600-compound library to identify existin...

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Pubblicato in:Hum Mol Genet
Autori principali: Sahdeo, Sunil, Scott, Brian D., McMackin, Marissa Z., Jasoliya, Mittal, Brown, Brandon, Wulff, Heike, Perlman, Susan L., Pook, Mark A., Cortopassi, Gino A.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2014
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Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4245046/
https://ncbi.nlm.nih.gov/pubmed/25113747
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu408
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