Zinc finger nuclease technology: advances and obstacles in modelling and treating genetic disorders
- PMID: 25536166
- DOI: 10.1016/j.gene.2014.12.044
Zinc finger nuclease technology: advances and obstacles in modelling and treating genetic disorders
Abstract
Zinc finger nucleases (ZFNs) are engineered restriction enzymes designed to target specific DNA sequences within the genome. Assembly of zinc finger DNA-binding domain to a DNA-cleavage domain enables the enzyme machinery to target unique locus in the genome and invoke endogenous DNA repair mechanisms. This machinery offers a versatile approach in allele editing and gene therapy. Here we discuss the architecture of ZFNs and strategies for generating targeted modifications within the genome. We review advances in gene therapy and modelling of the disease using these enzymes and finally, discuss the practical obstacles in using this technology.
Keywords: Disease modelling; Gene therapy; Zinc finger nuclease.
Copyright © 2014 Elsevier B.V. All rights reserved.
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