Human cystic fibrosis transmembrane conductance regulator directed to respiratory epithelial cells of transgenic mice

Nat Genet. 1992 Sep;2(1):13-20. doi: 10.1038/ng0992-13.


Human cystic fibrosis transmembrane conductance regulator (CFTR) was expressed in transgenic mice under the control of transcriptional elements derived from the human surfactant protein C (SP-C) gene. The hCFTR mRNA was expressed in lungs and testes: in the lung, we found hCFTR mRNA in bronchiolar and alveolar epithelial cells, and CFTR protein in respiratory epithelial cells. While the level of expression of hCFTR mRNA varied, hCFTR mRNA and protein were detected in pulmonary epithelial cells of several lines. Lung weight, morphology, somatic growth and reproductive capacity were not altered by expression hCFTR in lung and testes of the transgenics. Our findings suggest that hCFTR can be safely transferred to lung epithelial cells for CF therapy.

Publication types

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

MeSH terms

  • Animals
  • Cystic Fibrosis / genetics
  • Cystic Fibrosis / therapy
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Epithelium / metabolism
  • Gene Expression
  • Genetic Therapy
  • Humans
  • Immunohistochemistry
  • In Situ Hybridization
  • Lung / metabolism*
  • Male
  • Membrane Proteins / biosynthesis
  • Membrane Proteins / genetics*
  • Mice
  • Mice, Transgenic
  • Proteolipids / genetics
  • Pulmonary Surfactants / genetics
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Tissue Distribution


  • CFTR protein, human
  • Membrane Proteins
  • Proteolipids
  • Pulmonary Surfactants
  • RNA, Messenger
  • Cystic Fibrosis Transmembrane Conductance Regulator