|
|
|
题名
|
作者
|
年代
|
出处
|
被引量
|
| 1 | Homology-mediated end joining-based targeted integration using CRISPR/Cas9显示文摘 | Yao, Xuan Wang, Xing Hu, Xinde Liu, Zhen Liu, Junlai Zhou, Haibo Shen, Xiaowen Wei, Yu Huang, Zijian Ying, Wenqin Wang, Yan Nie, Yan-Hong Zhang, Chen-Chen Li, Sanlan Cheng, Leping Wang, Qifang Wu, Yan Huang, Pengyu Sun, Qiang Shi, Linyu Yang, Hui | 2017 | Cell Research2017，27，6： | 28 |
| 2 | 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 |
| 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 | CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration显示文摘RNA-targeting CRISPR system Cas13 offers an efficient approach for manipulating RNA transcripts in vitro.In this perspective,we provide a proof-of-concept demonstration that Cas13-mediated Vegfa knockdown in vivo could prevent the development of laser-induced CNV in mouse model of Age-related macular degeneration. | Changyang Zhou Xinde Hu Cheng Tang Wenjia Liu Shaoran Wang Yingsi Zhou Qimeng Zhao Qiyu Bo Linyu Shi Xiaodong Sun Haibo Zhou Hui Yang | 2020 | National Science Review2020，7，5： | 2 |
| 6 | 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 |
| 7 | Tild-CRISPR可在小鼠和人类细胞中实现高效、精确的基因敲入显示文摘文章简介研究团队基于CRISPR/Cas9系统,设计了一种新的靶向整合策略Tild-CRISPR,通过PCR扩增或者精确酶切,获得含有800bp同源臂的转基因供体与Cas9 mRNA以及single-guide RNA一起注射到小鼠受精卵中。 | 姚璇 Meiling Zhang Xing Wang Wenqin Ying Xinde Hu Pengfei Dai Feilong Meng Linyu Shi Yun Sun Ning Yao Wanxia Zhong Yun Li Keliang Wu 李卫平 陈子江 杨辉 | 2019 | 科学新闻2019，0，2： | 0 |
您所在的机构暂未开通此部分的功能，请联系管理员