piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice

Dis Model Mech. 2013 May;6(3):828-33. doi: 10.1242/dmm.010827. Epub 2013 Mar 8.


The stable genomic integration and expression of a large transgene is a major hurdle in gene therapy. We show that the modified piggyBac (PB) transposon system can be used to introduce a 207 kb genomic DNA fragment containing the RORγ/γt locus into human cells and mice. PB-mediated transgenesis results in a single copy of a stably inherited and expressed transgene. These results indicate that PB could serve as an effective high-capacity vector for functional analysis of the mammalian genome and for gene therapy in human cells.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Base Sequence
  • Chromosomes, Artificial, Bacterial / genetics
  • DNA Transposable Elements / genetics*
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / metabolism
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Genetic Vectors / genetics*
  • HEK293 Cells
  • Humans
  • Mice
  • Molecular Sequence Data
  • Mutagenesis, Insertional / genetics
  • Transgenes / genetics


  • DNA Transposable Elements