Rectal organoid-guided CFTR modulator therapy restores lung function in a cystic fibrosis patient with the rare 1677delTA/R334W genotype

Eur Respir J. 2022 Dec 15;60(6):2201341. doi: 10.1183/13993003.01341-2022. Print 2022 Dec.
No abstract available

Publication types

  • Letter
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Cystic Fibrosis* / drug therapy
  • Cystic Fibrosis* / genetics
  • Genotype
  • Humans
  • Lung
  • Mutation
  • Organoids

Substances

  • Cystic Fibrosis Transmembrane Conductance Regulator
  • CFTR protein, human