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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
Source PublicationCELL
AbstractGene-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 HeadingsScience & Technology ; Life Sciences & Biomedicine
Indexed BySCI
Funding OrganizationNational 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 Research AreaBiochemistry & Molecular Biology ; Cell Biology
WOS SubjectBiochemistry & Molecular Biology ; Cell Biology
WOS IDWOS:000401515900018
Citation statistics
Cited Times:71[WOS]   [WOS Record]     [Related Records in WOS]
Document Type期刊论文
Affiliation1.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
Recommended Citation
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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