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 RORγγ 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.
- Received September 4, 2012.
- Accepted March 6, 2013.
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