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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...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Hum Mol Genet |
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| Κύριοι συγγραφείς: | , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
Oxford University Press
2014
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| Θέματα: | |
| Διαθέσιμο 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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