PiggyBac transposon-based inducible gene expression in vivo after somatic cell gene transfer

Mol Ther. 2009 Dec;17(12):2115-20. doi: 10.1038/mt.2009.234. Epub 2009 Oct 6.

Abstract

Somatic cell gene transfer has permitted inducible gene expression in vivo through coinfection of multiple viruses. We hypothesized that the highly efficient plasmid-based piggyBac transposon system would enable long-term inducible gene expression in mice in vivo. We used a multiple-transposon delivery strategy to create a tetracycline-inducible expression system in vitro in human cells by delivering the two genes on separate transposons for inducible reporter gene expression along with a separate selectable transposon marker. Evaluation of stable cell lines revealed 100% of selected clones exhibited inducible expression via stable expression from three separate transposons simultaneously. We next tested and found that piggyBac-mediated gene transfer to liver or lung could achieve stable reporter gene expression in mice in vivo in either immunocompetent or immune deficient animals. A single injection of piggyBac transposons could achieve long-term inducible gene expression in the livers of mice in vivo, confirming our multiple-transposon strategy used in cultured cells. The plasmid-based piggyBac transposon system enables constitutive or inducible gene expression in vivo for potential therapeutic and biological applications without using viral vectors.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Blotting, Southern
  • Cells, Cultured
  • DNA Transposable Elements / genetics*
  • Female
  • Gene Transfer Techniques*
  • Genetic Vectors / administration & dosage*
  • Green Fluorescent Proteins / genetics
  • Green Fluorescent Proteins / metabolism*
  • Humans
  • Kidney / cytology
  • Kidney / metabolism
  • Liver / cytology
  • Liver / metabolism
  • Liver / virology
  • Lung / cytology
  • Lung / metabolism
  • Lung / virology
  • Mice
  • Mice, Inbred C57BL
  • Mice, SCID
  • Transgenes / genetics*
  • Virus Integration

Substances

  • DNA Transposable Elements
  • enhanced green fluorescent protein
  • Green Fluorescent Proteins