Directed evolution of adeno-associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells.

ABSTRACT: Efficient approaches for the precise genetic engineering of human pluripotent stem cells (hPSCs) can enhance both basic and applied stem cell research. Adeno- associated virus (AAV) vectors are of particular interest for their capacity to mediate efficient gene delivery to and gene targeting in various cells. However, natural AAV serotypes offer only modest transduction of human embryonic and induced pluripotent stem cells (hESCs and hiPSCs), which limits their utility for efficiently manipulating the hPSC genome. Directed evolution is a powerful means to generate viral vectors with novel capabilities, and we have applied this approach to create a novel AAV variant with high gene delivery efficiencies (~50%) to hPSCs, which are importantly accompanied by a considerable increase in gene-tar

SUBMITTER: Asuri P 

PROVIDER: S-EPMC3277219 | biostudies-literature | 2012 Feb

REPOSITORIES: biostudies-literature