piggyBac transposon-mediated long-term gene expression in mice.

piggyBac transposon-mediated long-term gene expression in mice.
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DOI:
10.1038/mt.2009.302
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发表时间:
2010-04
期刊:
Molecular therapy : the journal of the American Society of Gene Therapy
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Transposons are promising systems for somatic gene integration because they can not only integrate exogenous genes efficiently, but also be delivered to a variety of organs using a range of transfection methods. piggyBac (PB) transposon has a high transposability in mammalian cells in vitro, and has been used for genetic and preclinical studies. However, the transposability of PB in mammalian somatic cells in vivo has not been demonstrated yet. Here, we demonstrated PB-mediated sustained gene expression in adult mice. We constructed PB-based plasmid DNA (pDNA) containing reporter [firefly and Gaussia luciferase (Gluc)] genes. Mice were transfected by injection of these pDNAs using a hydrodynamics-based procedure, and the conditions for high-level sustained gene expression were examined. Consequently, gene expressions were sustained over 2 months. Our results suggest that PB is useful for organ-selective somatic integration and sustained gene expression in mammals, and will contribute to basic genetic studies and gene therapies.
使用由piggyBac转座子和聚乙烯亚胺组成的复合非病毒基因传递系统对小鼠进行肿瘤定向基因治疗
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