piggyBac-ing models and new therapeutic strategies

• The piggyBac transposon system offers a versatile genome modification system for biotechnology applications.
• piggyBac can be used for gene discovery or transgene insertion in vitro or in vivo.
• Stable cell lines can be more easily generated with piggyBac as compared with other gene delivery systems.
• New therapeutic strategies should result from piggyBac-related gene transfer research.

DNA transposons offer an efficient nonviral method of permanently modifying the genomes of mammalian cells. The piggyBac transposon system has proven effective in genomic engineering of mammalian cells for preclinical applications, including gene discovery, simultaneous multiplexed genome modification, animal transgenesis, gene transfer in vivo achieving long-term gene expression in animals, and the genetic modification of clinically relevant cell types, such as induced pluripotent stem cells and human T lymphocytes. piggyBac has many desirable features, including seamless excision of transposons from the genomic DNA and the potential to target integration events to desired DNA sequences. In this review, we explore these recent applications and also highlight the unique advantages of using piggyBac for developing new molecular therapeutic strategies.