【摘 要】
：
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 syst
【机 构】
：
State Key Laboratory of Genetic Engineering and Institute of Developmental Biology and Molecular Med
【出 处】
：
中国遗传学会第九次全国会员代表大会暨学术研讨会
论文部分内容阅读
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 highcapacity vector for functional analysis of the mammalian genome and for gene therapy in human cells.
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