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Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys
Chen, Yongchang1,3,14; Yu, Juehua2; Niu, Yuyu1,3,14; Qin, Dongdong5; Liu, Hailiang2; Li, Gang6,7; Hu, Yingzhou5; Wang, Jiaojian8; Lu, Yi9; Kang, Yu1,3,14; Jiang, Yong10,11; Wu, Kunhua10,11; Li, Siguang2; Wei, Jingkuan1,3; He, Jing1,3; Wang, Junbang2; Liu, Xiaojing2; Luo, Yuping2; Si, Chenyang1,3,14; Bai, Raoxian1,3; Zhang, Kunshan2; Liu, Jie2; Huang, Shaoyong1,3; Chen, Zhenzhen1,3; Wang, Shuang1,3; Chen, Xiaoying2; Bao, Xinhua12; Zhang, Qingping12; Li, Fuxing2; Geng, Rui2; Liang, Aibin2; Shen, Dinggang6,7; Jiang, Tianzi8,13; Hu, Xintian5; Ma, Yuanye1,3; Ji, Weizhi1,3,14; Sun, Yi Eve2,4
2017-05-18
发表期刊CELL
卷号169期号:5页码:945-+
文章类型Article
摘要Gene-editing technologies have made it feasible to create nonhuman primate models for human genetic disorders. Here, we report detailed genotypes and phenotypes of TALEN-edited MECP2 mutant cynomolgus monkeys serving as a model for a neurodevelopmental disorder, Rett syndrome (RTT), which is caused by loss-of-function mutations in the human MECP2 gene. Male mutant monkeys were embryonic lethal, reiterating that RTT is a disease of females. Through a battery of behavioral analyses, including primate-unique eye-tracking tests, in combination with brain imaging via MRI, we found a series of physiological, behavioral, and structural abnormalities resembling clinical manifestations of RTT. Moreover, blood transcriptome profiling revealed that mutant monkeys resembled RTT patients in immune gene dysregulation. Taken together, the stark similarity in phenotype and/or endophenotype between monkeys and patients suggested that gene-edited RTT founder monkeys would be of value for disease mechanistic studies as well as development of potential therapeutic interventions for RTT.
WOS标题词Science & Technology ; Life Sciences & Biomedicine
DOI10.1016/j.cell.2017.04.035
关键词[WOS]CPG-BINDING PROTEIN-2 ; NETWORK ANALYSIS ; MICE ; PRIMATES ; AUTISM ; GIRLS ; ABNORMALITIES ; NEUROANATOMY ; DEFICIENCY ; REGRESSION
收录类别SCI
语种英语
项目资助者National Key Research and Development Program(2016YFA0101401 ; National & Provincial Natural Science Foundation of China(U1602224 ; National Institute of Child Health Development of the NIH(U54HD087101-01) ; 2016YFA0100800) ; U1302227 ; 31571534 ; 2015FA037 ; 91319309 ; 31450110428 ; 31620103904 ; 2013HB133 ; 13JC1407102)
WOS研究方向Biochemistry & Molecular Biology ; Cell Biology
WOS类目Biochemistry & Molecular Biology ; Cell Biology
WOS记录号WOS:000401515900018
引用统计
文献类型期刊论文
条目标识符http://ir.ia.ac.cn/handle/173211/15097
专题脑网络组研究中心
作者单位1.Kunming Univ Sci & Technol, Inst Primate Translat Med, Yunnan Key Lab Primate Biomed Res, Kunming 650500, Peoples R China
2.Tongji Univ, Sch Med, Tongji Hosp, Translat Stem Cell Res Ctr, Shanghai 200065, Peoples R China
3.Yunnan Prov Acad Sci & Technol, Kunming 650051, Peoples R China
4.Univ Calif Los Angeles, Sch Med, Dept Psychiat & Biobehav Sci, Los Angeles, CA 90095 USA
5.Chinese Acad Sci, Kunming Inst Zool, Chinese Acad Sci & Yunnan Prov, Key Lab Anim Models & Human Dis Mech, Kunming 650223, Peoples R China
6.Univ North Carolina Chapel Hill, Dept Radiol, Chapel Hill, NC 27599 USA
7.Univ North Carolina Chapel Hill, BRIC, Chapel Hill, NC 27599 USA
8.Univ Elect Sci & Technol China, Sch Life Sci & Technol, Minist Educ, Key Lab NeuroInformat, Chengdu 625014, Peoples R China
9.Kunming Med Univ, Affiliated Hosp 1, Dept Med Imaging, Kunming 650032, Peoples R China
10.Kunming Univ Sci & Technol, Peoples Hosp Yunnan Prov 1, Kunming 650032, Peoples R China
11.Kunming Univ Sci & Technol, Affiliated Hosp, Kunming 650032, Peoples R China
12.Peking Univ, Hosp 1, Dept Pediat, Beijing 100034, Peoples R China
13.Chinese Acad Sci, Inst Automat, Brainnetome Ctr, Natl Lab Pattern Recognit, Beijing 100190, Peoples R China
14.Kunming Enovate Inst Biosci, Kunming 650000, Peoples R China
推荐引用方式
GB/T 7714
Chen, Yongchang,Yu, Juehua,Niu, Yuyu,et al. Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys[J]. CELL,2017,169(5):945-+.
APA Chen, Yongchang.,Yu, Juehua.,Niu, Yuyu.,Qin, Dongdong.,Liu, Hailiang.,...&Sun, Yi Eve.(2017).Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys.CELL,169(5),945-+.
MLA Chen, Yongchang,et al."Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys".CELL 169.5(2017):945-+.
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