1. Ali Zarei1,2*, Seyed Mohammad BagherTabei3, Vahid Razban4,5
Genome editing using CRISPR/Cas9 for creation animal and cell models of human disease
Ali Zarei1,2*, Seyed Mohammad BagherTabei3, Vahid Razban4,5  
1Department of Genetics, Fars Science and Research Branch, Islamic Azad University, Marvdasht, Iran.
2Department of Genetics, Marvdasht Branch, Islamic Azad University, Marvdasht, Iran.
3Department of Genetics, Shiraz University of Medical Science, Shiraz, Iran. 4Department of Molecular medicine, School of Advanced Medical Sciences and Technologies, Shiraz, Iran. 5Stem Cell and Transgenic Technology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran.
Introduction
Genome editing opens a new window for make clear a gene function that involved in a disease. There are different procedure using genome editing to clarify gene function, in which engineered nucleases are caused indel mutation in the genome. Until 2015 custom designed nucleases were classified into four family, including meganucleases, zinc finger nucleases (ZFNs), transcripition activator-like effector based nucleases (TALEN), and novel nuclease based gene editing system named clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas). All of the methods are to make a certain shift in a specific region of a gene.
Since human experimentation To find the underlying mechanism of human diseases would inconvenient or impractical, biomedical researcher extensively using animal models or cell line to imitate human disease, for instance mice that have same set of genes.
Essential goals of using animal and cell model are unveiled basis gene involvement process such as pathophysiological or molecular mechanism in genetics diseases and recognize novel candidate gene that causes both single gene or many gene disease.
Application of CRISPR/Cas9 to generate animal and cell model and its advantage
The benefits of CRISPR/Cas9 system compared to earlier method are simplicity, scalable and use of RNA gives a more accurate editing which make this editing system less expensive and time-consuming than ZFNs and TALENs.
References:
1. Turksen K. Genome Editing: Springer; p.
2. Cai M, Yang Y. Targeted genome editing tools for disease modeling and gene therapy. Current gene therapy. 2014;14(1):2-9.
3. He Z, Proudfoot C, Whitelaw CBA, Lillico SG. Comparison of CRISPR/Cas9 and TALENs on editing an integrated EGFP gene in the genome of HEK293FT cells. SpringerPlus. 2016;5(1):1-12.
4. Tschaharganeh DF, Lowe SW, Garippa RJ, Livshits G. Using CRISPR/Cas to study gene function and model disease in vivo. The FEBS journal. 2016;283(17):
5. Wolfe JH. Gene therapy in large animal models of human genetic diseases. Oxford University Press; 2009.
CRISPR/Cas9 system Compared with traditional gene targeting approaches has several profits, including simplicity of design, higher targeting efficiency and ability to create multiple mutation at the same time, which these benefits make CRISPR/Cas9 as a powerful genome engineering tool for instituting cell and animal model of human diseases.
Importance of producing animal and cell models of human diseases
Recently, The world of modern medical science with discovery of genome editing-mediated gene therapy method has deep changed.