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

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

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  • 1State Key Laboratory of Genetic Engineering and Institute of Developmental Biology and Molecular Medicine, Fudan-Yale Biomedical Research Center, School of Life Sciences, Fudan University, Shanghai 200433, China.


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.

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