Application of CRISPR-Cas system in gene therapy: Pre-clinical progress in animal model.

Lihong Guan, Yawei Han, Shaoyi Zhu, Juntang Lin

DNA repair 2016 Oct

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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. Copyright © 2016 Elsevier B.V. All rights reserved.

Citation

Lihong Guan, Yawei Han, Shaoyi Zhu, Juntang Lin. Application of CRISPR-Cas system in gene therapy: Pre-clinical progress in animal model. DNA repair. 2016 Oct;46:1-8