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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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Detalles Bibliográficos
Autores principales:	Sahdeo, Sunil, Scott, Brian D., McMackin, Marissa Z., Jasoliya, Mittal, Brown, Brandon, Wulff, Heike, Perlman, Susan L., Pook, Mark A., Cortopassi, Gino A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2014
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4245046/ https://www.ncbi.nlm.nih.gov/pubmed/25113747 http://dx.doi.org/10.1093/hmg/ddu408

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