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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2014
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Materias: | |
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 |