Article
Structure basis of CFTR folding, function and pharmacology.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2023
Hwang Tzyh-Chang, Braakman Ineke, van der Sluijs Peter, Callebaut Isabelle
Abstract excerpt
The root cause of cystic fibrosis (CF), the most common life-shortening genetic disease in the Caucasian population, is the loss of function of the CFTR protein, which serves as a phosphorylation-activated, ATP-gated anion channel in numerous epithelia-lining tissues. In the past decade, high-throughput drug screening has made a significant stride in developing highly effective CFTR modulators for the treatment...
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