Zinc finger nuclease technology: Advances and obstacles in modelling and treating genetic disorders

• An up-to-date review of Zinc finger nuclease technology in gene therapy and disease modelling
• A detailed overview of Zinc Finger endonucleases biochemistry
• Recent advances in modelling genetic diseases using ZFNs
• Recent development in the field of gene therapy using ZFNs

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.