Article
Allosteric inhibition of CFTR gating by CFTRinh-172 binding in the pore.
Nature communications - 6 Aug 2024
Gao Xiaolong, Yeh Han-I, Yang Zhengrong, Fan Chen, Jiang Fan, Howard Rebecca J, Lindahl Erik, Kappes John C, Hwang Tzyh-Chang
Abstract excerpt
Loss-of-function mutations of the CFTR gene cause the life-shortening genetic disease cystic fibrosis (CF), whereas overactivity of CFTR may lead to secretory diarrhea and polycystic kidney disease. While effective drugs targeting the CFTR protein have been developed for the treatment of CF, little progress has been made for diseases caused by hyper-activated CFTR. Here, we solve the cryo-EM structure of CFTR in...
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