Evaluating the potential for undesired genomic effects of the piggyBac transposon system in human cells.

ABSTRACT: Non-viral transposons have been used successfully for genetic modification of clinically relevant cells including embryonic stem, induced pluripotent stem, hematopoietic stem and primary human T cell types. However, there has been limited evaluation of undesired genomic effects when using transposons for human genome modification. The prevalence of piggyBac(PB)-like terminal repeat (TR) elements in the human genome raises concerns. We evaluated if there were undesired genomic effects of the PB transposon system to modify human cells. Expression of the transposase alone revealed no mobilization of endogenous PB-like sequences in the human genome and no increase in DNA double-strand breaks. The use of PB in a plasmid containing both transposase and transposon greatly increased the probabilit

SUBMITTER: Saha S 

PROVIDER: S-EPMC4330379 | biostudies-literature | 2015 Feb

REPOSITORIES: biostudies-literature