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Generation of nonhuman primate retinitis pigmentosa model by in situ knockout of RHO in rhesus macaque retina

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Pubmed体系：

收录情况： ◇ 统计源期刊 ◇ CSCD-C ◇ EI ◇ 卓越：领军期刊

作者：
Li Shouzhen[1] * ; Hu Yingzhou[2] * ; Li Yunqin[3] * ; Hu Min[3] ; Wang Wenchao[2] ; Ma Yuqian[1] ; Cai Yuan[1] ; Wei Min[1] ; Yao Yichuan[1] ; Wang Yun[4] ; Dong Kai[1] ; Gu Yonghao[1] ; Zhao Huan[6] ; Bao Jin[1] ; Qiu Zilong[5,8] ; Zhang Mei[1,10] ; Hu Xintian[2,4,5,9] ; Xue Tian[1,5,7,10] ;
机构： [1]Hefei National Laboratory for Physical Sciences at the Microscale, Eye Center at The First Affiliated Hospital of USTC, School of Life Sciences, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei 230026, China [2]Key Laboratory of Animal Models and Human Disease Mechanisms of Chinese Academy of Sciences & Yunnan Province, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming 650223, China [3]Second People’s Hospital of Yunnan Province, Yunnan Eye Institute, Key Laboratory of Yunnan Province for the Prevention and Treatment of Ophthalmology, Kunming 650223, China [4]Kunming Primate Research Center, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming 650223, China [5]Center for Excellence in Brain Science and Intelligence Technology, Chinese Academy of Sciences, Shanghai 200031, China [6]Department of Biological and Environmental Engineering, Hefei University, Hefei 230601, China [7]Institute for Stem Cell and Regeneration, Chinese Academy of Sciences, Beijing 100101, China [8]Institute of Neuroscience, State Key Laboratory of Neuroscience, Chinese Academy of Sciences, Shanghai 200031, China [9]National Research Facility for Phenotypic & Genetic Analysis of Model Animals (Primate Facility), Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming 650107, China [10]Neurodegenerative Disorder Research Center, CAS Key Laboratory of Brain Function and Disease, University of Science and Technology of China, Hefei 230026, China
出处：
DOI： 10.1016/j.scib.2020.09.008
ISSN：

关键词： Nonhuman primate model Retinitis pigmentosa Rhodopsin Disease model Gene editing SaCas9

摘要：
Retinitis pigmentosa (RP) is a form of inherited retinal degenerative diseases that ultimately involves the macula, which is present in primates but not in the rodents. Therefore, creating nonhuman primate (NHP) models of RP is of critical importance to study its mechanism of pathogenesis and to evaluate potential therapeutic options in the future. Here we applied adeno-associated virus (AAV)-delivered CRISPR/SaCas9 technology to knockout the RHO gene in the retinae of the adult rhesus macaque (Macaca mulatta) to investigate the hypothesis whether non-germline mutation of the RHO gene is sufficient to recapitulate RP. Through a series of studies, we were able to demonstrate successful somatic editing of the RHO gene and reduced RHO protein expression. More importantly, the mutant macaque retinae displayed clinical RP phenotypes, including photoreceptor degeneration, retinal thinning, abnormal rod subcellular structures, and reduced photoresponse. Therefore, we suggest somatic editing of the RHO gene is able to phenocopy RP, and the reduced time span in generating NHP mutant accelerates RP research and expands the utility of NHP model for human disease study.Copyright © 2020 Science China Press. Published by Elsevier B.V. All rights reserved.

基金：
This work was supported by the National Key Research and Development Program of China (2020YFA0112200, 2016YFA0400900, and 2018YFA0801403), the Strategic Priority Research Program of the Chinese Academy of Sciences (XDA16020603, XDB39000000, and XDB32060200), the National Natural Science Foundation of China (81925009, 81790644, 61890953, 31322024, 81371066, 91432104, 81900855, 31900712, and 31800901), Guangdong Provincial Key Research and Development Program (2019B030335001 and 2018B030338001), Anhui Provincial Natural Science Foundation (1808085MH289 and 1908085MC66), and the Fundamental Research Funds for the Central Universities (WK2070000174 and WK2090050048).
语种：
外文
PubmedID：
中科院(CAS)分区：
出版当年[2021]版：
大类 | 1 区 综合性期刊
小类 | 1 区 综合性期刊
最新[2023]版：
大类 | 1 区 综合性期刊
小类 | 1 区 综合性期刊
第一作者：
第一作者机构： [1]Hefei National Laboratory for Physical Sciences at the Microscale, Eye Center at The First Affiliated Hospital of USTC, School of Life Sciences, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei 230026, China
共同第一作者：
通讯作者：
通讯机构： [1]Hefei National Laboratory for Physical Sciences at the Microscale, Eye Center at The First Affiliated Hospital of USTC, School of Life Sciences, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei 230026, China [2]Key Laboratory of Animal Models and Human Disease Mechanisms of Chinese Academy of Sciences & Yunnan Province, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming 650223, China [4]Kunming Primate Research Center, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming 650223, China [5]Center for Excellence in Brain Science and Intelligence Technology, Chinese Academy of Sciences, Shanghai 200031, China [7]Institute for Stem Cell and Regeneration, Chinese Academy of Sciences, Beijing 100101, China [9]National Research Facility for Phenotypic & Genetic Analysis of Model Animals (Primate Facility), Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming 650107, China [10]Neurodegenerative Disorder Research Center, CAS Key Laboratory of Brain Function and Disease, University of Science and Technology of China, Hefei 230026, China
推荐引用方式(GB/T 7714)：
Li Shouzhen,Hu Yingzhou,Li Yunqin,et al.Generation of nonhuman primate retinitis pigmentosa model by in situ knockout of RHO in rhesus macaque retina[J].Science Bulletin.2021,66(4):374-385.doi:10.1016/j.scib.2020.09.008.
APA：
Li Shouzhen,Hu Yingzhou,Li Yunqin,Hu Min,Wang Wenchao...&Xue Tian.(2021).Generation of nonhuman primate retinitis pigmentosa model by in situ knockout of RHO in rhesus macaque retina.Science Bulletin,66,(4)
MLA：
Li Shouzhen,et al."Generation of nonhuman primate retinitis pigmentosa model by in situ knockout of RHO in rhesus macaque retina".Science Bulletin 66..4(2021):374-385

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