title: 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.
authors.affiliation: School of Veterinary Medicine, Azad University of Shahrekord, Shahrekord, Iran.
authors.forename: Hamid Reza
authors.initials: HR
authors.lastname: Jabalameli
authors.affiliation: Institute of Biochemistry and Biophysics, University of Tehran, Tehran, Iran.
authors.forename: Hamid
authors.initials: H
authors.lastname: Zahednasab
authors.affiliation: Division of Genetics, Department of Biology, School of Science, University of Isfahan, Isfahan, Iran.
authors.forename: Amin
authors.initials: A
authors.lastname: Karimi-Moghaddam
authors.affiliation: Human Genetics and Genomic Medicine, Faculty of Medicine, University of Southampton, Southampton General Hospital, Southampton, UK.
authors.forename: Mohammad Reza
authors.initials: MR
authors.lastname: Jabalameli
mesh_terms: D000818:Animals; D004260:DNA Repair; D004720:Endonucleases / Q000235:genetics*; D018390:Gene Targeting; D005818:Genetic Engineering; D015316:Genetic Therapy; D006801:Humans; D016335:Zinc Fingers
chemical_list: D004720:Endonucleases
keywords: Disease modelling; Gene therapy; Zinc finger nuclease