title: Application of CRISPR-Cas system in gene therapy: Pre-clinical progress in animal model.
abstract: The clustered regularly interspaced short palindromic repeats (CRISPRs) and their associated proteins (Cas) belong to the crucial adaptive immune system, which exist in archaea and bacteria. Currently, CRISPR-Cas9 system has been modified and widely used to edit genome. In this review, we summarized the discovery, classification and mechanism of CRISPR-Cas system and further discussed the application of CRISPR-Cas9 in gene therapy, mainly in disease models.
authors: College of Life Science and Technology, Xinxiang Medical University, Xinxiang, Henan, 453003, China; Henan Key Laboratory of Medical Tissue Regeneration, Xinxiang, Henan, 453003, China.
authors: Lihong
authors: L
authors: Guan
authors: College of Food and Bioengineering, Zhengzhou University of Light Industry, Zhengzhou, Henan, 450002, China.
authors: Yawei
authors: Y
authors: Han
authors: College of Life Science and Technology, Xinxiang Medical University, Xinxiang, Henan, 453003, China.
authors: Shaoyi
authors: S
authors: Zhu
authors: College of Life Science and Technology, Xinxiang Medical University, Xinxiang, Henan, 453003, China; Henan Key Laboratory of Medical Tissue Regeneration, Xinxiang, Henan, 453003, China; College of Biomedical Engineering, Xinxiang Medical University, Xinxiang, Henan, 453003, China. Electronic address: linjtlin@126.com.
authors: Juntang
authors: J
authors: Lin
mesh_terms: D000163:Acquired Immunodeficiency Syndrome / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D000230:Adenocarcinoma / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D000077192:Adenocarcinoma of Lung; D000818:Animals; D064113:CRISPR-Cas Systems; D002386:Cataract / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D004195:Disease Models, Animal; D000072669:Gene Editing / Q000379:methods; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D008175:Lung Neoplasms / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D009765:Obesity / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D000069896:Transcription Activator-Like Effector Nucleases / Q000235:genetics / Q000378:metabolism; D020176:Tyrosinemias / Q000235:genetics / Q000473:pathology / Q000628:therapy*
chemical_list: D000069896:Transcription Activator-Like Effector Nucleases
keywords: CRISPR-Cas9; DNA repair; Disease model; Gene therapy; Genome editing