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J Gene Med. 2019 Feb 20:e3082. doi: 10.1002/jgm.3082. [Epub ahead of print]

Creating cell and animal models of human disease by genome editing using CRISPR/Cas9.

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Department of Molecular Genetics, Marvdasht Branch, Islamic Azad University, Marvdasht, Iran.
Department of Molecular Genetics, Science and Research Branch, Islamic Azad University, Fars, Iran.
Department of Molecular medicine, School of Advanced Medical Sciences and Technologies, Shiraz, Iran.
Stem Cell and Transgenic Technology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran.
Department of Physiology, Marvdasht Branch, Islamic Azad University, Marvdasht, Iran.
Department of Genetics, Shiraz University of Medical Science, Shiraz, Iran.


A set of unique sequences in bacterial genomes, responsible for protecting bacteria against bacteriophages, has recently been used for the genetic manipulation of specific points in the genome. These systems consist of one RNA component and one enzyme component, known as CRISPR ("clustered regularly interspaced short palindromic repeats") and Cas9, respectively. The present review focuses on the applications of CRISPR/Cas9 technology in the development of cellular and animal models of human disease. Making a desired genetic alteration depends on the design of RNA molecules that guide endonucleases to a favorable genomic location. With the discovery of CRISPR/Cas9 technology, researchers are able to achieve higher levels of accuracy because of its advantages over alternative methods for editing genome, including a simple design, a high targeting efficiency and the ability to create simultaneous alterations in multiple sequences. These factors allow the researchers to apply this technology to creating cellular and animal models of human diseases by knock-in, knock-out and Indel mutation strategies, such as for Huntington's disease, cardiovascular disorders and cancers. Optimized CRISPR/Cas9 technology will facilitate access to valuable novel cellular and animal genetic models with respect to the development of innovative drug discovery and gene therapy.


animal model; gene delivery; gene editing; transfection


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