CFTR: New insights into structure and function and implications for modulation by small molecules - Sorbonne Université
Journal Articles Journal of Cystic Fibrosis Year : 2020

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

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.
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Dates and versions

hal-03030412 , version 1 (30-11-2020)

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Bertrand Kleizen, John Hunt, Isabelle Callebaut, Tzyh-Chang Hwang, Isabelle Sermet-Gaudelus, et al.. CFTR: New insights into structure and function and implications for modulation by small molecules. Journal of Cystic Fibrosis, 2020, 19 (S1), pp.S19-S24. ⟨10.1016/j.jcf.2019.10.021⟩. ⟨hal-03030412⟩
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