Coexpression of the uracil phosphoribosyltransferase gene with a chimeric human nerve growth factor receptor/cytosine deaminase fusion gene, using a single retroviral vector, augments cytotoxicity of transduced human T cells exposed to 5-fluorocytosine

Hum Gene Ther. 2006 May;17(5):518-30. doi: 10.1089/hum.2006.17.518.

Abstract

Donor T lymphocytes genetically engineered to express a "suicide gene" to facilitate negative selection represent a promising strategy for the management of graft-versus-host disease occurring after allogeneic hematopoietic cell transplantation (HCT). For this purpose, the herpes simplex virus thymidine kinase (HSV-tk) gene, although well studied, has limitations. Cytosine deaminase (CD), an alternative gene for negative selection, converts 5-fluorocytosine (5-FC) to the toxic metabolite 5-fluorouracil (5-FU). Sensitivity of cells to 5-FU can be further increased by expression of uracil phosphoribosyltransferase (UPRT), which catalyzes the conversion of 5-FU to 5-fluorouridine monophosphate. By using a chimeric gene (NG/CD) expressing the truncated human nerve growth factor receptor (NGFR) for positive selection fused to the Saccharomyces cerevisiae CD gene, we investigated strategies to achieve optimal T cell eradication by CD and UPRT expression, utilizing a single retroviral vector. Three vector strategies were compared on the basis of NGFR expression by flow cytometry, western analysis, and enzymatic activity. A construct (NG/CDiU) expressing UPRT and NG/CD, using a bicistronic message, provided the greatest UPRT activity and killing, reducing the lethal dose of 5-FC sufficient to eradicate 90% of cells from 38.7 microg/ml (300 microM) (NG/CD expression alone) to 0.13 microg/ml (1 microM). This approach provides an effective alternative to the HSV-tk system for eradication of donor T lymphocytes after allogeneic HCT.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line
  • Cell Proliferation
  • Cytosine Deaminase / genetics*
  • Escherichia coli / genetics
  • Flucytosine / toxicity*
  • Genes, Transgenic, Suicide*
  • Genetic Vectors
  • Humans
  • Pentosyltransferases / genetics*
  • Pentosyltransferases / metabolism
  • Pentosyltransferases / toxicity
  • Pyrimidines / metabolism
  • Pyrimidines / toxicity
  • Receptor, Nerve Growth Factor / genetics*
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / toxicity
  • Retroelements / genetics
  • Saccharomyces cerevisiae / genetics
  • T-Lymphocytes*
  • Transduction, Genetic / methods

Substances

  • Pyrimidines
  • Receptor, Nerve Growth Factor
  • Recombinant Fusion Proteins
  • Retroelements
  • 5-fluoropyrimidine
  • Flucytosine
  • Pentosyltransferases
  • uracil phosphoribosyltransferase
  • Cytosine Deaminase