哺乳动物的初始编辑:下一代精确基因组编辑。

IF 3.7 4区 生物学 Q2 GENETICS & HEREDITY CRISPR Journal Pub Date : 2022-12-01 DOI:10.1089/crispr.2022.0084
Dawei Wang, Xiude Fan, Mengzhu Li, Tianbo Liu, Peng Lu, Guangxin Wang, Yuan Li, JunMing Han, JiaJun Zhao
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引用次数: 0

摘要

最近建立的初始编辑器(PE)系统被认为是下一代基因编辑技术。这种方法可以安装任何碱基到碱基的变化以及插入和删除,而不需要双链断裂形成或供体DNA模板;因此,它比传统的CRISPR-Cas系统或碱基编辑器提供了更大的靶向灵活性和更高的编辑精度。在这项研究中,我们介绍了PE的基本原理,然后回顾了其在哺乳动物中编辑的多功能性、特异性和效率方面的最新进展。接下来,我们总结了PE变体的选择、启动编辑指导RNA (pegRNA)设计规则以及PE的效率和准确性评估方面的关键考虑因素。最后,我们强调并讨论了PE如何协助广泛的生物学研究,以及如何应用PE在动物模型中进行精确的基因组校正,从而为治疗人类疾病铺平道路。
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Prime Editing in Mammals: The Next Generation of Precision Genome Editing.

The recently established prime editor (PE) system is regarded as next-generation gene-editing technology. This methodology can install any base-to-base change as well as insertions and deletions without the requirement for double-stranded break formation or donor DNA templates; thus, it offers more targeting flexibility and greater editing precision than conventional CRISPR-Cas systems or base editors. In this study, we introduce the basic principles of PE and then review its most recent progress in terms of editing versatility, specificity, and efficiency in mammals. Next, we summarize key considerations regarding the selection of PE variants, prime editing guide RNA (pegRNA) design rules, and the efficiency and accuracy evaluation of PE. Finally, we highlight and discuss how PE can assist in a wide range of biological studies and how it can be applied to make precise genomic corrections in animal models, which paves the way for curing human diseases.

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来源期刊
CRISPR Journal
CRISPR Journal Biochemistry, Genetics and Molecular Biology-Biotechnology
CiteScore
6.30
自引率
2.70%
发文量
76
期刊介绍: In recognition of this extraordinary scientific and technological era, Mary Ann Liebert, Inc., publishers recently announced the creation of The CRISPR Journal -- an international, multidisciplinary peer-reviewed journal publishing outstanding research on the myriad applications and underlying technology of CRISPR. Debuting in 2018, The CRISPR Journal will be published online and in print with flexible open access options, providing a high-profile venue for groundbreaking research, as well as lively and provocative commentary, analysis, and debate. The CRISPR Journal adds an exciting and dynamic component to the Mary Ann Liebert, Inc. portfolio, which includes GEN (Genetic Engineering & Biotechnology News) and more than 80 leading peer-reviewed journals.
期刊最新文献
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