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

Publication date: Available online 30 July 2016
Source:DNA Repair
Author(s): Lihong Guan, Yawei Han, Shaoyi Zhu, Juntang Lin
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.



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