College of Tobacco Science and Engineering, Zhengzhou University of Light Industry, Zhengzhou, 450002, Henan, China.
Stem Cells and Biotherapy Engineering Research Center of Henan, National Joint Engineering Laboratory of Stem Cells and Biotherapy, School of Life Science and Technology, Xinxiang Medical University, Number 601, Jinsui Road, Xinxiang, 453003, Henan, China.
Mol Genet Genomics. 2024 Aug 1;299(1):75. doi: 10.1007/s00438-024-02166-x.
The clustered regularly interspaced short palindromic repeats (CRISPR) and their associated protein (Cas) system is a gene editing technology guided by RNA endonuclease. The CRISPR-Cas12a (also known as CRISPR-Cpf1) system is extensively utilized in genome editing research due to its accuracy and high efficiency. In this paper, we primarily focus on the application of CRISPR-Cpf1 technology in the construction of disease models and gene therapy. Firstly, the structure and mechanism of the CRISPR-Cas system are introduced. Secondly, the similarities and differences between CRISPR-Cpf1 and CRISPR-Cas9 technologies are compared. Thirdly, the main focus is on the application of the CRISPR-Cpf1 system in cell and animal genome editing. Finally, the challenges faced by CRISPR-Cpf1 technology and corresponding strategies are analyzed. Although CRISPR-Cpf1 technology has certain off-target effects, it can effectively and accurately edit cell and animal genomes, and has significant advantages in the preclinical research.
簇状规律间隔短回文重复序列（CRISPR）及其相关蛋白（Cas）系统是一种由 RNA 内切酶指导的基因编辑技术。CRISPR-Cas12a（也称为 CRISPR-Cpf1）系统由于其准确性和高效率，在基因组编辑研究中得到了广泛应用。本文主要关注 CRISPR-Cpf1 技术在疾病模型构建和基因治疗中的应用。首先，介绍了 CRISPR-Cas 系统的结构和机制。其次，比较了 CRISPR-Cpf1 和 CRISPR-Cas9 技术的异同。再次，重点介绍了 CRISPR-Cpf1 系统在细胞和动物基因组编辑中的应用。最后，分析了 CRISPR-Cpf1 技术面临的挑战及相应策略。虽然 CRISPR-Cpf1 技术存在一定的脱靶效应，但它可以有效地、精确地编辑细胞和动物基因组，在临床前研究中具有显著优势。
