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.
Keywords:
CRISPR-Cas9; DNA repair; Disease model; Gene therapy; Genome editing.
Copyright © 2016 Elsevier B.V. All rights reserved.
MeSH terms
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Acquired Immunodeficiency Syndrome / genetics
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Acquired Immunodeficiency Syndrome / pathology
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Acquired Immunodeficiency Syndrome / therapy*
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Adenocarcinoma / genetics
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Adenocarcinoma / pathology
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Adenocarcinoma / therapy*
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Adenocarcinoma of Lung
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Animals
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CRISPR-Cas Systems*
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Cataract / genetics
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Cataract / pathology
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Cataract / therapy*
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Disease Models, Animal
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Gene Editing / methods
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Genetic Therapy / methods
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Humans
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Lung Neoplasms / genetics
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Lung Neoplasms / pathology
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Lung Neoplasms / therapy*
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Mice
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Muscular Dystrophy, Duchenne / genetics
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Muscular Dystrophy, Duchenne / pathology
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Muscular Dystrophy, Duchenne / therapy*
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Obesity / genetics
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Obesity / pathology
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Obesity / therapy*
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Transcription Activator-Like Effector Nucleases / genetics
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Transcription Activator-Like Effector Nucleases / metabolism
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Tyrosinemias / genetics
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Tyrosinemias / pathology
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Tyrosinemias / therapy*
Substances
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Transcription Activator-Like Effector Nucleases