Zinc Finger Nucleases: Tailor-made for Gene Therapy.

ABSTRACT: Genome editing with the use of zinc finger nucleases has been successfully applied to variety of a eukaryotic cells. Furthermore, the proof of concept for this approach has been extended to diverse animal models from Drosophila to mice. Engineered zinc finger nucleases are able to target specifically and manipulate disease-causing genes through site-specific double strand DNA breaks followed by non-homologous end joining or homologous recombination mechanisms. Consequently, this technology has considerable flexibility that can result in either a gain or loss of function of the targeted gene. In addition to this flexibility, gene therapy by zinc finger nucleases may enable persistent long term gene modification without continuous transfection- a potential advantage over RNA interfere

SUBMITTER: Chou ST 

PROVIDER: S-EPMC3801298 | biostudies-literature | 2012 Mar

REPOSITORIES: biostudies-literature