Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes

Mol Ther. 2002 Aug;6(2):199-209. doi: 10.1006/mthe.2002.0653.

Abstract

Gene therapy is an attractive approach for the treatment of liver disease. We demonstrate that a so-called third-generation human immunodeficiency virus (HIV)-derived vector system can govern the efficient delivery, integration, and stable expression of a transgene into primary human hepatocytes in the complete absence of cell division. We also show that rodent hepatocytes exhibit a significant degree of resistance to HIV vector-mediated transduction, a phenotype that is particularly pronounced in murine hepatocytes and that results from a block in the immediate-early phase of infection. We finally describe a methodology, that allows very high rates of transduction through minimal in vitro manipulation, in which hepatocytes are kept in suspension and reimplanted within a few hours of harvest with a fully preserved engraftment potential. These results have immediate implications for the treatment of liver diseases by the transplantation of genetically modified hepatocytes, an approach that could be applied to a number of hereditary and acquired hepatic disorders.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Division
  • Cell Line
  • Gene Transfer Techniques
  • Genetic Therapy / methods
  • Genetic Vectors*
  • HIV / genetics
  • HeLa Cells
  • Hepatocytes / cytology*
  • Hepatocytes / transplantation
  • Humans
  • In Vitro Techniques
  • Lentivirus / genetics*
  • Liver Diseases / therapy
  • Male
  • Mice
  • Rats
  • Rats, Sprague-Dawley
  • Transduction, Genetic*