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. 2020 Mar:19 Suppl 1:S19-S24.
doi: 10.1016/j.jcf.2019.10.021. Epub 2019 Nov 21.

CFTR: New insights into structure and function and implications for modulation by small molecules

Bertrand Kleizen  1 , John F Hunt  2 , Isabelle Callebaut  3 , Tzyh-Chang Hwang  4 , Isabelle Sermet-Gaudelus  5 , Sylvia Hafkemeyer  6 , David N Sheppard  7
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Free article

CFTR: New insights into structure and function and implications for modulation by small molecules

Bertrand Kleizen et al. J Cyst Fibros. 2020 Mar.
Free article

Abstract

Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives.

Keywords: ABC-transporter; Clinical drugs; Cystic fibrosis; F508del; Membrane protein; Mutations; Therapy.

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Conflict of interest statement

Declaration of Competing Interest DNS is the recipient of a Vertex Innovation Award, IS-G is the recipient of two Vertex Innovation Awards and a member of the scientific boards of Eloxx, PTC Therapeutics and Vertex Therapeutics. T-CH has an ongoing service agreement with Abbvie and a sponsored research grant from Nanova.

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