Correction of the apical membrane chloride permeability defect in polarized cystic fibrosis airway epithelia following retroviral-mediated gene transfer.

摘要: ABSTRACT We are studying the introduction and expression of normal cystic fibrosis transmembrane conductance regulator (CFTR) cDNA into cultured human airway epithelial cells as a model for gene therapy fibrosis. In this paper, we show that chloride transport defect at apical membrane is corrected in vitro differentiated ion-transporting CF exhibit polarized properties similar to those found vivo. Using retroviral vector containing copy CFTR cDNA, infected cultures proliferating, CFT1 correction was maintained following differentiation sheet. At least partial Cl¯ preserved periods up 6 months without selection maintenance provirus. These results suggest it may be feasible target proliferating lung using vectors treatment disease.