Article
Towards next generation therapies for cystic fibrosis: Folding, function and pharmacology of CFTR.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2020
Bose Samuel J, Krainer Georg, Ng Demi R S, Schenkel Mathias, Shishido Hideki, Yoon Jae Seok, Haggie Peter M, Schlierf Michael, Sheppard David N, Skach William R
Abstract excerpt
The treatment of cystic fibrosis (CF) has been transformed by orally-bioavailable small molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR), which restore function to CF mutants. However, CFTR modulators are not available to all people with CF and better modulators are required to prevent disease progression. Here, we review selectively recent advances in CFTR folding, function...
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