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

Bertrand Kleizen*, John Hunt, Isabelle Callebaut, Tzyh-Chang Hwang, Isabelle Sermet-Gaudelus, Sylvia Hafkemeyer, David N Sheppard

*Corresponding author for this work

Research output: Contribution to journalArticle (Academic Journal)peer-review

6 Citations (Scopus)
29 Downloads (Pure)

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.
Original languageEnglish
Pages (from-to)S19-S24
Number of pages6
JournalJournal of Cystic Fibrosis
Volume19
Issue numberSupplement 1
Early online date21 Nov 2019
DOIs
Publication statusPublished - Mar 2020

Keywords

  • membrane protein
  • ABC-transporter
  • mutations
  • F508del
  • therapy
  • clinical drugs
  • cystic fibrosis

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