Article
Phenotypic Screening for Friedreich Ataxia Using Random shRNA Selection.
Journal of biomolecular screening - 1 Oct 2015
Cotticelli M Grazia, Acquaviva Fabio, Xia Shujuan, Kaur Avinash, Wang Yongping, Wilson Robert B
Abstract excerpt
Friedreich ataxia (FRDA) is an autosomal recessive neuro- and cardio-degenerative disorder for which there are no proven effective treatments. FRDA is caused by decreased expression and/or function of the protein frataxin. Frataxin chaperones iron in the mitochondrial matrix and regulates the iron-sulfur cluster (ISC) assembly complex. ISCs are prosthetic groups critical for the function of the Krebs cycle and...
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