2 fold, either as a vector-expressed shRNA or as a transfected siRNA. We randomly mutagenized gFA2 to create a gFA2 variant sub-library. We screened this sub-library in primary FA fibroblasts and identified two gFA2 variants,...
Lin, Daniel, Tobias, John W., Cotticelli, M. Gracia, Ruff, Eric, Wang, Yongping, Xia, Shujuan, Wilson, Robert B., and Kaur, Avinash
Beschreibung:
Friedreich ataxia (FRDA) is an autosomal recessive neuro- and cardio-degenerative disorder caused by decreased expression of frataxin, a protein that localizes to mitochondria and is critical for iron-sulfur-cluster (ISC)...