单核苷酸编辑:从原理、优化到应用。
Single-nucleotide editing: From principle, optimization to application.
机构信息
Center for Precision Medicine, School of Medicine and School of Biomedical Sciences, Huaqiao University, Xiamen, Fujian, China.
Department of Cell and Developmental Biology, Weill Medical College, Cornell University, New York, New York.
出版信息
Hum Mutat. 2019 Dec;40(12):2171-2183. doi: 10.1002/humu.23819. Epub 2019 Sep 15.
Cytosine base editors (CBEs) and adenine base editors (ABEs), which are generally composed of an engineered deaminase and a catalytically impaired CRISPR-Cas9 variant, are new favorite tools for single base substitution in cells and organisms. In this review, we summarize the principle of base-editing systems and elaborate on the evolution of different platforms of CBEs and ABEs, including their deaminase, Cas9 variants, and editing outcomes. Moreover, we highlight their applications in mouse and human cells and discuss the challenges and prospects of base editors. The ABE- and CBE systems have been used in gene silencing, pathogenic gene correction, and functional genetic screening. Single base editing is becoming a new promising genetic tool in biomedical research and gene therapy.
胞嘧啶碱基编辑器 (CBEs) 和腺嘌呤碱基编辑器 (ABEs) 通常由工程化的脱氨酶和催化失活的 CRISPR-Cas9 变体组成,是细胞和生物体内单碱基替换的新工具。在这篇综述中,我们总结了碱基编辑系统的原理,并详细阐述了不同 CBEs 和 ABEs 平台的发展,包括它们的脱氨酶、Cas9 变体和编辑结果。此外,我们还强调了它们在小鼠和人类细胞中的应用,并讨论了碱基编辑器的挑战和前景。ABE 和 CBE 系统已被用于基因沉默、致病基因校正和功能遗传筛选。单碱基编辑正在成为生物医学研究和基因治疗中的一种新的有前途的遗传工具。
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