Effective Targeted Gene Knockdown in Mammalian Cells Using the piggyBac Transposase-based Delivery System.

ABSTRACT: Nonviral gene delivery systems are rapidly becoming a desirable and applicable method to overexpress genes in various types of cells. We have recently developed a piggyBac transposase-based, helper-independent and self-inactivating delivery system (pmGENIE-3) capable of high-efficiency transfection of mammalian cells including human cells. In the following study, we have assessed the potential of this delivery system to drive the expression of short hairpin RNAs to knock down genes in human cells. Two independent pmGENIE-3 vectors were developed to specifically target knockdown of an endogenous gene, telomerase reverse transcriptase (TERT), in telomerase-positive human immortalized cell lines. As compared with a transposase-deficient vector, pmGENIE-3 showed significantly improved short-te

SUBMITTER: Owens JB 

PROVIDER: S-EPMC3894583 | biostudies-literature | 2013 Dec

REPOSITORIES: biostudies-literature