High lysosomal activities in cystic fibrosis tracheal gland cells corrected by adenovirus-mediated CFTR gene transfer

Biochim Biophys Acta. 1999 Jan 6;1453(1):14-22. doi: 10.1016/s0925-4439(98)00084-2.

Abstract

Human tracheal gland serous (HTGS) cells are now believed to be a major target of cystic fibrosis (CF) gene therapy. To evaluate the efficiency of adenovirus-mediated gene transfer in these cells we tested the adenovirus construction containing beta-galactosidase cDNA. We observed that the endogenous beta-galactosidase activity in cultured CF-HTGS cells was too strong to allow us to detect any exogenous beta-galactosidase activity. Immunohistological study on sections of human tracheal tissue confirmed the presence of beta-galactosidase in the serous component of the submucosal glands. We then looked for other lysosomal activities in normal and CF-HTGS cells. We showed that normal cells already have elevated enzyme values and that CF-HTGS cells contained 2-4-fold more beta-galactosidase, alpha-fucosidase, alpha-mannosidase and beta-glucuronidase activities than normal cells. An analysis of their kinetic constants has shown that this difference could be attributed to a lower K(m) of CF lysosomal enzymes. More importantly, these differences are eliminated after adenovirus-mediated CFTR gene transfer and not after beta-galactosidase gene transfer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cells, Cultured
  • Cystic Fibrosis / enzymology
  • Cystic Fibrosis / therapy
  • Cystic Fibrosis Transmembrane Conductance Regulator / biosynthesis
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
  • Gene Transfer Techniques
  • Genetic Therapy
  • Genetic Vectors
  • Humans
  • Immunohistochemistry
  • In Vitro Techniques
  • Lysosomes / enzymology*
  • Trachea / enzymology*
  • Trachea / ultrastructure
  • beta-Galactosidase / biosynthesis
  • beta-Galactosidase / genetics

Substances

  • CFTR protein, human
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • beta-Galactosidase