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    题名 作者 年代 出处 被引量
1Efficient base editing in G/C-rich regions to model androgen insensitivity syndrome显示文摘Dear Editor, Most disease-associated genomic mutations are base substitutions and approximately half of pathogenic human single nucleotide polymorphisms (SNPs)are related to C-to-T substitutions in the ClinVar database.Base editors (BEs),which combine Casg-DIOA nickase and APOBEC (apolipoprotein B mRNA editing enzyme,catalytic polypeptide-like)or AID (activation-induced deaminase)cytidine deaminase family members, have been successfully applied to mediate C-to-T conversion in vitro and in vivo, providing a powerful tool to model or repair diseaserelated human SNPs.Jianan Li Zhen Liu Shisheng Huang Xiao Wang Guanglei Li Yuting Xu Wenxia Yu Shanshan Chen Yu Zhang Hanhui Ma Zunfu Ke Jia Chen Qiang Sun Xingxu Huang 2019Cell Research2019,29,2:3
2Efficient and high-fidelity base editor with expanded PAM compatibility for cytidine dinucleotide显示文摘Cytidine base editor(CBE),which is composed of a cytidine deaminase fused to Cas9 nickase,has been widely used to induce C-to-T conversions in a wide range of organisms.However,the targeting scope of current CBEs is largely restricted to protospacer adjacent motif(PAM)sequences containing G,T,or A bases.In this study,we developed a new base editor termed“nNme2-CBE”with excellent PAM compatibility for cytidine dinucleotide,significantly expanding the genome-targeting scope of CBEs.Using nNme2-CBE,targeted editing efficiencies of 29.0%-55.0%and 17.3%—52.5%were generated in human cells and rabbit embryos,respectively.In contrast to conventional nSp-CBE,the nNme2-CBE is a natural high-fidelity base editing platform with minimal DNA off-targeting detected in vivo.Significantly increased efficiency in GC context and precision were determined by combining nNme2Cas9 with rationally engineered cytidine deaminases.In addition,the Founder rabbits with accurate single-base substitutions at Fgf5 gene loci were successfully generated by using the nNme2-CBE system.These novel nNme2-CBEs with expanded PAM compatibility and high fidelity will expand the base editing toolset for efficient gene modification and therapeutic applications.Zhiquan Liu Siyu Chen Yingqi Jia Huanhuan Shan Mao Chen Yuning Song Liangxue Lai Zhanjun Li 2021Science China(Life Sciences)2021,64,8:2
3First progeria monkey model generated using base editor显示文摘In this issue,Wang et al.report on the generation of a nonhuman primate model of Hutchinson-Gilford progeria syndrome(HGPS)using a base editor.Base editing is an emerging novel genome editing technique for modifying a single base pair at specific sites in the genome.Base editors(BEs)have two prin cipal comp orients,a catalytically in active or single strand cleaving Cas-variant,which binds to the guide RNA and a nucleobase deaminase domain to convert specific base pairs at the target loci(Komor et al.,2016;Nishida et al.,2016;Gaudelli et al.,2017).Cytosine base editor(CBE)and adenine base editor(ABE)are two baseeditors,which con vert Cytosi ne-Gua nine(C-G)to Thymine-Adenine(T-A)and A-T to G-C,respectively.Pradeep Reddy Yanjiao Shao Reyna Hernandez-Benitez Estrella Nunez Delicado Juan Carlos Izpisua Belmonte 2020Protein & Cell2020,11,12:1
4CRISPR base editor treats premature-aging syndrome显示文摘A recent paper published in Nature by Koblan et al.reported the use of CRISPR-mediated adenine base editor(ABE)to repair mutations of the Hutchinson-Gilford progeria syndrome(HGPS or progeria),attenuate symptoms,and extend lifespan of mice(Fig.1),representing a major advance in design of treatments for human accelerated-ageing disorders and potentially other genetic diseases.Ping Lin Jianxin Jiang Min Wu 2021Signal Transduction and Targeted Therapy2021,6,5:0
5Engineering a plant A-to-K base editor with improved performance by fusion with a transactivation module显示文摘Dear Editor,Base editors(BEs)are promising tools that enable single-nucleotide substitutions in specific target genes,generating loss-offunction or gain-of-function mutations,which can greatly accelerate plant functional genomics and crop improvement(Ren et al.,2018;Zeng et al.,2020;Xu et al.,2021;Yan et al.,2021;Li et al.,2023).To date,three major classes of BEs have been engineered:cytosine BEs(CBEs)for C-to-T transitions(Komor et al.,2016),adenine BEs(ABEs)for A-to-G transitions(Gaudelli et al.,2017).Yucai Li Shaoya Li Chenfei Li Chen Zhang Lei Yan Jingying Li Yubing He Yan Guo Yong Lin Yangjun Zhang Lanqin Xia 2023Plant Communications2023,4,6:0
6Adenine base editor incorporating the N-methylpurine DNA glycosylase MPGv3 enables efficient A-to-K base editing in rice显示文摘Dear Editor,Crop genetic diversity and elite agronomic traits are mainly caused by genetic variants,approximately half of which are single-nucleotide polymorphisms.Precise nucleotide substitution through CRISPR–Cas-mediated base editors has been widely used to correct defective alleles and create novel alleles by artificial evolution for rapid crop genetic improvement.Since 2017,cytosine base editors(CBEs)and adenine base editors(ABEs)have been successively developed in many plant species and have been continuously optimized to generate highly efficient C-to-T and A-to-G transitions,as well as by-product C-to-A/G conversion with low efficiency(Ren et al.,2018;Yan et al.,2021).Xuemei Wu Bin Ren Lang Liu Shengqun Qiu Xin’ge Li Peijing Li Fang Yan Honghui Lin Xueping Zhou Dawei Zhang Huanbin Zhou 2023Plant Communications2023,4,6:0
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