Restoration of superoxide generation to a chronic granulomatous disease-derived B-cell line by retrovirus mediated gene transfer

Blood. 1992 Sep 1;80(5):1125-9.

Abstract

Failure of a superoxide generating system, the NADPH oxidase, present in neutrophils and other phagocytes gives rise to chronic granulomatous disease (CGD), a group of single-gene inherited disorders all characterized by an extreme susceptibility to pyogenic infection, with potentially fatal consequences. About 30% of CGD cases are caused by an autosomally inherited deficiency of a 47-Kd cytoplasmic component of the oxidase (p47-phox). Epstein-Barr virus (EBV) immortalized B-lymphocyte lines established from these CGD patients also express this NADPH oxidase defect and consequently are rendered incapable of generating superoxide on stimulation. We have used a p47-phox-deficient EBV-transformed B-cell line as a recipient for retroviral transfer of a functional p47-phox cDNA. The presence and activity of the retrovirally encoded p47-phox in the transduced cells is demonstrated and we show that this restores their capacity to generate superoxide.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • B-Lymphocytes / metabolism*
  • Cell Line
  • Genetic Therapy
  • Granulomatous Disease, Chronic / metabolism
  • Granulomatous Disease, Chronic / therapy*
  • Herpesvirus 4, Human / genetics*
  • Humans
  • NADH, NADPH Oxidoreductases / genetics*
  • NADPH Oxidases
  • Superoxides / metabolism*
  • Transfection*

Substances

  • Superoxides
  • NADH, NADPH Oxidoreductases
  • NADPH Oxidases