Engineering nucleic acid chemistry for precise and controllable CRISPR/Cas9 genome editing

Weiqi Cai; Ming Wang

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Engineering nucleic acid chemistry for precise and controllable CRISPR/Cas9 genome editing

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The clustered regularly interspaced short palindromic repeats(CRISPR)/associated protein 9(CRISPR/Cas9) genome editing technology is revolutionizing our approach and capability to precisely manipulate the genetic flow of mammalians. The facile programmability of Cas9 protein and guide RNA(g RNA)sequence has recently expanded biomedical application of CRISPR/Cas9 technology from editing mammalian genome to various genetic manipulations. The therapeutic and clinical translation potential of CRISPR/Cas9 genome editing, however, are challenged by its off-target effect and low genome editing efficiency. In this regard, developing new Cas9 variants and conditional control of Cas9/g RNA activity are of great potential for improving genome editing accuracy and on-target efficiency. In this review, we summarize chemical strategies that have been developed recently to engineer the nucleic acid chemistry of g RNA to enhance CRISPR/Cas9 genome editing efficacy, specificity and controllability. This review aims to highlight the endeavor that has been made to solve bottleneck problems in the field of CRISPR/Cas9 and inspire innovative researches to fulfill the gap between bench and bed.

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《中国科学通报：英文版》 |2019年第24期|1841-1849|共9页
作者
Weiqi Cai; Ming Wang;
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作者单位

Beijing National Laboratory for Molecular Sciences;

Key Laboratory of Analytical Chemistry for Living Biosystems;

CAS Research/Education Center for Excellence in Molecule Science;

Institute of Chemistry;

Chinese Academy of Sciences;

Beijing 100190;

China;

University of Chinese Academy of Sciences;

Beijing 100049;

China;

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原文格式 PDF
正文语种 chi
中图分类基因工程（遗传工程）;
关键词
CRISPR/Cas9 genome editing; RNA engineering; Nucleic acid chemistry; Gene therapy; Aptamer;

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Engineering nucleic acid chemistry for precise and controllable CRISPR/Cas9 genome editing

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