A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon.

ABSTRACT: Nonviral vector systems are used increasingly in gene targeting and gene transfer applications. The piggyBac transposon represents an alternative integrating vector for in vivo gene transfer. We hypothesized that this system could achieve persistent gene transfer to the liver when administered systemically. We report that a novel hyperactive transposase generated higher transposition efficiency than a codon-optimized transposase in a human liver cell line. Hyperactive transposase-mediated reporter gene expression persisted at levels twice that of codon-optimized transposase in the livers of mice for the 6-month study. Of note, expression persisted in mice following partial hepatectomy, consistent with expression from an integrated transgene. We also used the hyperactive transposase to deli

SUBMITTER: Burnight ER 

PROVIDER: S-EPMC3499692 | biostudies-literature | 2012 Oct

REPOSITORIES: biostudies-literature