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

作者：Guan Lihong; Han Yawei; Zhu Shaoyi; Lin Juntang

来源：DNA Repair, 2016, 46: 1-8.

DOI：10.1016/j.dnarep.2016.07.004

• CRISPR-Cas9
• Genome editing
• Disease model
• DNA repair
• Gene therapy

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.

• 出版日期2016-10
• 单位郑州轻工业大学; 新乡医学院