Gene therapy for cystic fibrosis: new tools for precision medicine

J Transl Med. 2021 Oct 30;19(1):452. doi: 10.1186/s12967-021-03099-4.


The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada. Recent developments in gene targeting tools and new cell and animal models have re-ignited the search for a permanent genetic cure for all CF. In this review, we highlight some of the more recent gene therapy approaches as well as new models that will provide insight into personalized therapies for CF.

Keywords: Alternative chloride channels; CFTR; Cystic fibrosis; Lung; Organoids; Precision medicine; Stem cells; TMEM16A.

Publication types

  • Review

MeSH terms

  • Animals
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Cystic Fibrosis* / genetics
  • Cystic Fibrosis* / therapy
  • Genetic Therapy
  • Humans
  • Middle Aged
  • Mutation
  • Precision Medicine


  • Cystic Fibrosis Transmembrane Conductance Regulator