title: 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 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.
authors.affiliation: State 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.
authors.forename: Rongbo
authors.initials: R
authors.lastname: Li
authors.forename: Yuan
authors.initials: Y
authors.lastname: Zhuang
authors.forename: Min
authors.initials: M
authors.lastname: Han
authors.forename: Tian
authors.initials: T
authors.lastname: Xu
authors.forename: Xiaohui
authors.initials: X
authors.lastname: Wu
mesh_terms: D000818:Animals; D001483:Base Sequence; D022202:Chromosomes, Artificial, Bacterial / Q000235:genetics; D004251:DNA Transposable Elements / Q000235:genetics*; D053595:Embryonic Stem Cells / Q000166:cytology / Q000378:metabolism; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics*; D057809:HEK293 Cells; D006801:Humans; D051379:Mice; D008969:Molecular Sequence Data; D016254:Mutagenesis, Insertional / Q000235:genetics; D019076:Transgenes / Q000235:genetics
chemical_list: D004251:DNA Transposable Elements