Hepatoma cell-specific ganciclovir-mediated toxicity of a lentivirally transduced HSV-TkEGFP fusion protein gene placed under the control of rat alpha-fetoprotein gene regulatory sequences

Cancer Gene Ther. 2003 Sep;10(9):689-95. doi: 10.1038/sj.cgt.7700621.

Abstract

Suicide gene therapy combining herpes simplex virus thymidine kinase gene transfer and ganciclovir administration can be envisioned as a powerful therapeutical approach in the treatment of hepatocellular carcinoma; however, safety issues regarding transgene expression in parenchyma cells have to be addressed. In this study, we constructed LATKW, a lentiviral vector expressing the HSV-TkEGFP gene placed under the control of the promoter elements that control the expression of the rat alpha-fetoprotein, and assayed its specific expression in vitro in hepatocarcinoma and nonhepatocarcinoma human cell lines, and in epidermal growth factor stimulated human primary hepatocytes. Using LATKW, a strong expression of the transgene was found in transduced hepatocarcinoma cells compared to a very low expression in nonhepatocarcinoma human cell lines, as assessed by Northern blot, RT-PCR, FACS analysis and ganciclovir-mediated toxicity assay, and no expression was found in lentivirally transduced normal human hepatocytes. Altogether, these results demonstrate the possibility to use a lentivirally transduced expression unit containing the rat alpha-fetoprotein promoter to restrict the HSV-TK-mediated induced GCV sensitivity to human hepatocarcinoma cells.

MeSH terms

  • Animals
  • Blotting, Northern
  • Carcinoma, Hepatocellular / genetics
  • Carcinoma, Hepatocellular / metabolism
  • Carcinoma, Hepatocellular / pathology*
  • Carcinoma, Hepatocellular / therapy*
  • Cell Death
  • Cell Line, Tumor
  • Ganciclovir / toxicity*
  • Genetic Therapy*
  • Genetic Vectors / genetics
  • Green Fluorescent Proteins
  • Hepatocytes / cytology
  • Hepatocytes / metabolism
  • Hepatocytes / pathology
  • Humans
  • Lentivirus / genetics
  • Luminescent Proteins / genetics
  • Luminescent Proteins / metabolism
  • Microscopy, Fluorescence
  • Organ Specificity
  • Promoter Regions, Genetic / genetics
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Rats
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / metabolism
  • Thymidine Kinase / genetics*
  • Thymidine Kinase / metabolism*
  • Transduction, Genetic
  • Transfection
  • alpha-Fetoproteins / genetics*

Substances

  • Luminescent Proteins
  • RNA, Messenger
  • Recombinant Fusion Proteins
  • alpha-Fetoproteins
  • Green Fluorescent Proteins
  • Thymidine Kinase
  • Ganciclovir