Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis

Cell. 1993 Oct 22;75(2):207-16. doi: 10.1016/0092-8674(93)80063-k.


To evaluate the potential of direct transfer of cystic fibrosis transmembrane conductance regulator (CFTR) cDNA for the treatment of cystic fibrosis (CF), we administered an E1-deficient adenovirus, encoding CFTR, to a defined area of nasal airway epithelium of three individuals with CF. This treatment corrected the Cl- transport defect that is characteristic of CF-affected epithelia. After treatment, there was a decrease in the elevated basal transepithelial voltage, and the normal response to a cAMP agonist was restored. We found no evidence of viral replication or virus-associated adverse effects, even at the highest dose tested (25 MOI). These data represent a small step in achieving long-term improvement of CF lung function by gene therapy.

Publication types

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

MeSH terms

  • Adenoviridae / genetics
  • Adult
  • Amiloride / pharmacology
  • Biological Transport
  • Chlorides / metabolism*
  • Cystic Fibrosis / genetics
  • Cystic Fibrosis / therapy*
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Epithelium / metabolism
  • Female
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Humans
  • Male
  • Membrane Potentials / drug effects
  • Membrane Proteins / genetics*
  • Middle Aged
  • Nasal Mucosa / metabolism*
  • Nasal Mucosa / pathology
  • Safety
  • Terbutaline / pharmacology


  • CFTR protein, human
  • Chlorides
  • Membrane Proteins
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Amiloride
  • Terbutaline