title: Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell.
abstract: After 25 years of intensive search, there is not yet a cure for cystic fibrosis (CF). However, the quest has led to major breakthroughs in understanding the basic disease defect and defining strategies to correct it. The first cystic fibrosis transmembrane conductance regulator (CFTR) modulators have been introduced in clinic. Some show an impressive clinical benefit, like the potentiator ivacaftor for the 4% of patients with a class III defect. Others offer at present only a limited benefit, like the combination corrector lumacaftor plus potentiator ivacaftor for subjects homozygous for F508del. These findings prove that the basic defect in CF can be modified and hold the promise that one day CF will no longer be a life-shortening disease.
  
  
  CONCLUSIONS
  This review updates the clinician on recent achievements as well as on the CF research pipeline.
  
  
  BACKGROUND
  Cystic fibrosis (CF) is a common and life-shortening disease that currently cannot be cured. However, for each of the six CF mutation classes, disease-modifying drugs are under way.
  
  
  BACKGROUND
  This review is a concise update for the clinician on new drugs that reached the CF clinical pipeline. The research strategies in CF have become a paradigm for clinical trials in other inherited diseases.
authors.affiliation: Department of Paediatric Pulmonology, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium. barbara.bosch@uzleuven.be.
authors.forename: Barbara
authors.initials: B
authors.lastname: Bosch
authors.affiliation: Department of Paediatric Pulmonology, University Hospitals Leuven, Herestraat 49, 3000, Leuven, Belgium. christiane.deboeck@uzleuven.be.
authors.forename: Kris
authors.initials: K
authors.lastname: De Boeck
mesh_terms: D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics / Q000502:physiology; D015316:Genetic Therapy; D006801:Humans; D014176:Protein Biosynthesis; D012333:RNA, Messenger / Q000235:genetics
chemical_list: C505032:CFTR protein, human; D012333:RNA, Messenger; D019005:Cystic Fibrosis Transmembrane Conductance Regulator
keywords: CFTR; Clinical trials; Corrector; Cystic fibrosis; N-of-1 trial; Organoids; Potentiator