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

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Li, R; Zhuang, Y; Han, M; Xu, T; Wu, X

Published Date

  • May 2013

Published In

Volume / Issue

  • 6 / 3

Start / End Page

  • 828 - 833

PubMed ID

  • 23519027

Pubmed Central ID

  • PMC3634665

Electronic International Standard Serial Number (EISSN)

  • 1754-8411

Digital Object Identifier (DOI)

  • 10.1242/dmm.010827


  • eng

Conference Location

  • England