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

ABSTRACT: 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 ROR?/?t 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.

SUBMITTER: Li R

PROVIDER: S-EPMC3634665 | biostudies-literature | 2013 May

REPOSITORIES: biostudies-literature

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

Li Rongbo R Zhuang Yuan Y Han Min M Xu Tian T Wu Xiaohui X

Disease models & mechanisms 20130308 3

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 ...[more]

PMID: 23519027

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