Corrector combination therapies for F508del-CFTR

Curr Opin Pharmacol. 2017 Jun;34:105-111. doi: 10.1016/j.coph.2017.09.016. Epub 2017 Nov 5.


These are exciting times in the development of therapeutics for cystic fibrosis (CF). New correctors and potentiators of the cystic fibrosis transmembrane conductance regulator (CFTR) are being developed in academic laboratories and pharmaceutical companies, and the field is just beginning to understand their mechanisms of action. Studies of CFTR modulators are also yielding insight into the general principles and strategies that can be used when developing pharmacological chaperones, a new class of drugs. Combining two or even three correctors with a potentiator is an especially promising approach which should lead to further improvements in efficacy and clinical benefit for patients.

Publication types

  • Review

MeSH terms

  • Aminophenols / pharmacology
  • Binding Sites
  • Chloride Channel Agonists / pharmacology
  • Chloride Channel Agonists / therapeutic use
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / metabolism
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Cystic Fibrosis Transmembrane Conductance Regulator / metabolism*
  • Drug Therapy, Combination
  • Humans
  • Mutation
  • Quinolones / pharmacology


  • Aminophenols
  • CFTR protein, human
  • Chloride Channel Agonists
  • Quinolones
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • ivacaftor

Grant support