| Induction of core symptoms of autism spectrum disorder by in vivo CRISPR/Cas9-based gene editing in the brain of adolescent rhesus monkeys |
| Wu, SH; Li, X ; Qin, DD; Zhang, LH; Cheng, TL; Chen, ZF; Nie, BB; Ren, XF; Wu, J; Wang, WC; Hu, YZ; Gu, YL; Lv, LB; Yin, Y; Hu, XT; Qiu, ZL
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| 2021
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| 发表期刊 | SCIENCE BULLETIN
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| ISSN | 2095-9273
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| 卷号 | 66期号:9页码:937-946 |
| 摘要 | Although CRISPR/Cas9-mediated gene editing is widely applied to mimic human disorders, whether acute manipulation of disease-causing genes in the brain leads to behavioral abnormalities in non-human primates remains to be determined. Here we induced genetic mutations in MECP2, a critical gene linked to Rett syndrome (RTT) and autism spectrum disorders (ASD), in the hippocampus (DG and CA1?4) of adolescent rhesus monkeys (Macaca mulatta) in vivo via adeno-associated virus (AAV)-delivered Staphylococcus aureus Cas9 with small guide RNAs (sgRNAs) targeting MECP2. In comparison to monkeys injected with AAV-SaCas9 alone (n = 4), numerous autistic-like behavioral abnormalities were identified in the AAV-SaCas9-sgMECP2-injected monkeys (n = 7), including social interaction deficits, abnormal sleep patterns, insensitivity to aversive stimuli, abnormal hand motions, and defective social reward behaviors. Furthermore, some aspects of ASD and RTT, such as stereotypic behaviors, did not appear in the MECP2 gene-edited monkeys, suggesting that different brain areas likely contribute to distinct ASD symptoms. This study showed that acute manipulation of disease-causing genes via in vivo gene editing directly led to behavioral changes in adolescent primates, paving the way for the rapid generation of genetically engineered non-human primate models for neurobiological studies and therapeutic development. ? 2020 Science China Press. Published by Elsevier B.V. and Science China Press. All rights reserved. |
| 收录类别 | sci
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| 语种 | 英语
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| 文献类型 | 期刊论文
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| 条目标识符 | http://ir.kiz.ac.cn/handle/152453/13269
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| 专题 | 科研部门_神经系统编码（胡新天）
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推荐引用方式
GB/T 7714 |
Wu, SH,Li, X,Qin, DD,et al. Induction of core symptoms of autism spectrum disorder by in vivo CRISPR/Cas9-based gene editing in the brain of adolescent rhesus monkeys[J]. SCIENCE BULLETIN,2021,66(9):937-946.
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| APA |
Wu, SH.,Li, X.,Qin, DD.,Zhang, LH.,Cheng, TL.,...&Qiu, ZL.(2021).Induction of core symptoms of autism spectrum disorder by in vivo CRISPR/Cas9-based gene editing in the brain of adolescent rhesus monkeys.SCIENCE BULLETIN,66(9),937-946.
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| MLA |
Wu, SH,et al."Induction of core symptoms of autism spectrum disorder by in vivo CRISPR/Cas9-based gene editing in the brain of adolescent rhesus monkeys".SCIENCE BULLETIN 66.9(2021):937-946.
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