Prolonged activity of the transposase helper may raise safety concerns during DNA transposon-based gene therapy.

ABSTRACT: DNA transposon-based gene delivery vectors represent a promising new branch of randomly integrating vector development for gene therapy. For the side-by-side evaluation of the piggyBac and Sleeping Beauty systems-the only DNA transposons currently employed in clinical trials-during therapeutic intervention, we treated the mouse model of tyrosinemia type I with liver-targeted gene delivery using both transposon vectors. For genome-wide mapping of transposon insertion sites we developed a new next-generation sequencing procedure called streptavidin-based enrichment sequencing, which allowed us to identify approximately one million integration sites for both systems. We revealed that a high proportion of piggyBac integrations are clustered in hot regions and found that th

SUBMITTER: Imre G 

PROVIDER: S-EPMC10070360 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature