Disease Models & Mechanisms (May 2013)

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

  • Rongbo Li,
  • Yuan Zhuang,
  • Min Han,
  • Tian Xu,
  • Xiaohui Wu

DOI
https://doi.org/10.1242/dmm.010827
Journal volume & issue
Vol. 6, no. 3
pp. 828 – 833

Abstract

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SUMMARY 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.