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| 1 | One-step generation of complete gene knockout mice and monkeys by CRISPR/Cas9-mediated gene editing with multiple sgRNAs显示文摘CRISPR/Cas9 系统是一个有效编辑基因的方法,但是编辑基因的动物的多数显示出 mosaicism,与编辑仅仅在房间的部分发生。这里,我们证明那单个基因或多重基因能被 Cas9 mRNA 和多重邻近的单个指南的 RNA 的 zygotic 注射完全在老鼠和猴子胚胎击倒(spaced 10-200 bp 分开) 指向仅仅一把单个钥匙每基因的 exon。个别地在 Y 染色体上跟随八基因的指向的删除的 F0 老鼠的 Phenotypic 分析在产生猛烈老鼠表明了这条途径的坚韧性。重要地,这条途径在高效率交付完全的基因大美人(Arntl 上的 100% 并且 91% 在 Prrt2 上) 在猴子胚胎。最后,我们能在一个单个步骤产生一只完全的 Prrt2 猛烈猴子,表明在很快建立编辑基因的猴子模型的这条途径的实用性。 | Erwei Zuo Yi-Jun Cai Kui Li Yu Wei Bang-An Wang Yidi Sun Zhen Liu Jiwei Liu Xinde Hu Wei Wei Xiaona Huo Linyu Shi Cheng Tang Dan Liang Yan Wang Yan-Hong Nie Chen-Chen Zhang Xuan Yao Xing Wang Changyang Zhou Wenqin Ying Qifang Wang Ren-Chao Chen Qi Shen Guo-Liang Xu Jinsong Li Qiang Sun Zhi-Qi Xiong Hui Yang | 2017 | Cell Research2017，27，7： | 21 |
| 2 | CRISPR-Cas9-mediated genome editing in one blastomere of two-cell embryos reveals a novel Tet3 function in regulating neocortical development显示文摘学习必要基因的早功能是在发展生物学的一个重要、挑战性的问题。这里,我们为很快在二房间的阶段胚胎的一个分裂球导致 CRISPR-Cas9-mediated 变化建立了一个方法,称为的 2 房间 embryo-CRISPR-Cas9 注射(2CC ) ,学习在里面必需品的 vivo 功能(或未知) 在创始人的基因妄想的鼠标。由注射指向兴趣的基因进的 Cre mRNA 和 CRISPR-Cas9 荧光灯记者老鼠, 2CC 方法能在不同发展阶段跟踪野类型、变异的房间,为变异的房间的 phenotypic 分析的出现内部控制。用这个方法,我们在在发展中的鼠标调整有刺激性、禁止的 synaptic 传播识别了必要基因 Tet3 的新奇函数服的外皮。由产生妄想的变异的老鼠, 2CC 方法允许在多重纸巾和房间的基因功能的快速的屏蔽录入妄想的老鼠创始人,显著地扩展基因工具的当前的一套设备。 | Wang, Lingbo Li, Min-Yin Qu, Chao Miao, Wan-Ying Yin, Qi Liao, Jiaoyang Cao, Hua-Teng Huang, Min Wang, Kai Zuo, Erwei Peng, Guangdun Zhang, Shu-Xin Chen, Guodong Li, Qing Tang, Ke Yu, Qian Li, Zhoujie Wong, Catherine C. L. Xu, Guoliang Jing, Naihe Yu, Xiang Li, Jinsong | 2017 | Cell Research2017，27，6： | 5 |
| 3 | Base editing-mediated splicing correction therapy for spinal muscular atrophy显示文摘Dear Editor,Spinal muscular atrophy(SMA)is a devastating autosomal recessive motor neuron disease.1,2 Infants with more severe forms of type I SMA die before the age of 2 if no intervention is provided.1,2 Spinraza and zolgensma have been approved by the FDA as SMA therapeutics for pediatric patients.1,2 However,as an antisense oligonucleotide(ASO)based therapy,spinraza requires four loading doses,followed by three annual maintenance doses.The patients would be subjected to repeated intrathecal injections in the procedure.Zolgensma is a single-dose gene-replacement therapy for SMA,but unfortunately is unreliable in maintaining a high,stable level of gene expression.These issues have limited the therapeutic effects of the two approved drugs for SMA. | Xiang Lin Haizhu Chen Ying-Qian Lu Shunyan Hong Xinde Hu Yanxia Gao Lu-Lu Lai Jin-Jing Li Zishuai Wang Wenqin Ying Lixiang Ma Ning Wang Erwei Zuo Hui Yang Wan-Jin Chen | 2020 | Cell Research2020，30，6： | 5 |
| 4 | Treatment of autosomal recessive hearing loss via in vivo CRISPR/Cas9-mediated optimized homology-directed repair in mice显示文摘Dear Editor,Hearing loss is the most common sensory disorder in the world.Among cases of non-syndromic hearing loss,which account for 70%of all cases of genetic hearing loss,around 80%of cases arise from autosomal recessive loss-of-function mutations that require repair,rather than disruption,of the mutant allele.1 CRISPR/Cas9-mediated homology-directed repair(HDR)-based therapies have the potential to cure many genetic diseases because this class of therapeutics can achieve arbitrary base changes as well as the insertion or deletion of DNA fragments/However. | Xi Gu Xinde Hu Daqi Wang Zhijiao Xu Fang Wang Di Li Geng-lin Li Hui Yang Huawei Li Erwei Zuo Yilai Shu | 2022 | Cell Research2022，32，7： | 3 |
| 5 | The advancements,challenges,and future implications of the CRISPR/Cas9 system in swine research显示文摘Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9(CRISPR/Cas9)genome editing technology has dramatically influenced swine research by enabling the production of high-quality disease-resistant pig breeds,thus improving yields.In addition,CRISPR/Cas9 has been used extensively in pigs as one of the tools in biomedical research.In this review,we present the advancements of the CRISPR/Cas9 system in swine research,such as animal breeding,vaccine development,xenotransplantation,and disease modeling.We also highlight the current challenges and some potential applications of the CRISPR/Cas9 technologies. | Jinfu Zhang Emmanuel M.Khazalwa Hussein M.Abkallo Yuan Zhou Xiongwei Nie Jinxue Ruan Changzhi Zhao Jieru Wang Jing Xu Xinyun Li Shuhong Zhao Erwei Zuo Lucilla Steinaa Shengsong Xie | 2021 | Journal of Genetics and Genomics2021，48，5： | 2 |
| 6 | Indiscriminate ssDNA cleavage activity of CRISPR-Cas12a induces no detectable off-target effects in mouse embryos显示文摘Dear Editor,Newly discovered characteristics like'collateral effect'or trans-cleavage in CRISPR-Cas13 and CRISPR-Cas12 systems have enabled their usage in nucleic acid detection(Gootenberg et al.2017,2018;Chen et al.2018).The collateral RNA cleavage of Cas13a has been reported to be harmful for cell development(Wang et al.2019;Buchman et al.2020).As a representative gene editor of CRISPR-Cas12 system,CRISPR-Cas12a(Cpf1)holds great potential for therapeutic applications in the future(Zetsche et al.2015;Koo et al.2018;Campa et al.2019). | Yu Wei Yingsi Zhou Yajing Liu Wenqin Ying Ruiming Lv Qimeng Zhao Haibo Zhou Erwei Zuo Yidi Sun Hui Yang Changyang Zhou | 2021 | Protein & Cell2021，12，9： | 2 |
| 7 | Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and mice显示文摘We here report a genome-editing strategy to correct spinal muscular atrophy(SMA).Rather than directly targeting the pathogenic exonic mutations,our strategy employed Cas9 and guide-sg RNA for the targeted disruption of intronic splicing-regulatory elements.We disrupted intronic splicing silencers(ISSs,including ISS-N1 and ISS+100)of survival motor neuron(SMN)2,a key modifier gene of SMA,to enhance exon 7 inclusion and full-length SMN expression in SMA iPSCs.Survival of splicing-corrected iPSC-derived motor neurons was rescued with SMN restoration.Furthermore,co-injection of Cas9 mRNA from Streptococcus pyogenes(SpCas9)or Cas9 from Staphylococcus aureus(SaCas9)alongside their corresponding sgRNAs targeting ISS-N1 into zygotes rescued 56%and 100%of severe SMA transgenic mice(Smn-/-,SMN2tg/-).The median survival of the resulting mice was extended to>400 days.Collectively,our study provides proof-of-principle for a new strategy to therapeutically intervene in SMA and other RNA-splicing-related diseases. | Jin-Jing Li Xiang Lin Cheng Tang Ying-Qian Lu Xinde Hu Erwei Zuo He Li Wenqin Ying Yidi Sun Lu-Lu Lai Hai-Zhu Chen Xin-Xin Guo Qi-Jie Zhang Shuang Wu Changyang Zhou Xiaowen Shen Qifang Wang Min-Ting Lin Li-Xiang Ma Ning Wang Adrian R.Krainer Linyu Shi Hui Yang Wan-Jin Chen | 2020 | National Science Review2020，7，1： | 1 |
| 8 | Human 8-cell embryos enable efficient induction of disease-preventive mutations without off-target effect by cytosine base editor显示文摘Approximately 140 million people worldwide are homozygous carriers of APOE4(ε4),a strong genetic risk factor for late onset familial and sporadic Alzheimer’s disease(AD),91%of whom will develop AD at earlier age than heterozygous carriers and noncarriers.Susceptibility to AD could be reduced by targeted editing of APOE4,but a technical basis for controlling the off-target effects of base editors is necessary to develop low-risk personalized gene therapies.Here,we first screened eight cytosine base editor variants at four injection stages(from 1-to 8-cell stage),and found that FNLS-YE1 variant in 8-cell embryos achieved the comparable base conversion rate(up to 100%)with the lowest bystander effects.In particular,80%of AD-susceptibleε4 allele copies were converted to the AD-neutralε3 allele in humanε4-carrying embryos.Stringent control measures combined with targeted deep sequencing,whole genome sequencing,and RNA sequencing showed no DNA or RNA off-target events in FNLS-YE1-treated human embryos or their derived stem cells.Furthermore,base editing with FNLS-YE1 showed no effects on embryo development to the blastocyst stage.Finally,we also demonstrated FNLS-YE1 could introduce known protective variants in human embryos to potentially reduce human susceptivity to systemic lupus erythematosus and familial hypercholesterolemia.Our study therefore suggests that base editing with FNLS-YE1 can efficiently and safely introduce known preventive variants in 8-cell human embryos,a potential approach for reducing human susceptibility to AD or other genetic diseases. | Yinghui Wei Meiling Zhang Jing Hu Yingsi Zhou Mingxing Xue Jianhang Yin Yuanhua Liu Hu Feng Ling Zhou Zhifang Li Dongshuang Wang Zhiguo Zhang Yin Zhou Hongbin Liu Ning Yao Erwei Zuo Jiazhi Hu Yanzhi Du Wen Li Chunlong Xu Hui Yang | 2023 | Protein & Cell2023，14，6： | 0 |
| 9 | Base editing technology显示文摘The largest class of known human pathogenic mutations is point mutation,also known as single nucleotide polymorphism(SNP)[1].Precise and efficient editing of these SNPs has been of great interest for the treatment of genetic disorders.Base editing is a genome editing method which can precisely induce point mutations on DNA or RNA at the target loci without generating double strand breaks[2–5]. | Yidi SUN Erwei ZUO Hui YANG | 2020 | Frontiers of Agricultural Science and Engineering2020，7，2： | 0 |
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