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CasRx-mediated RNA targeting prevents choroidal neovascularization in a 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
doi: https://doi.org/10.1101/2020.02.18.955286
Changyang Zhou
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
2College of Life Sciences, University of Chinese Academy of Sciences, Beijing 100049, China
Xinde Hu
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
2College of Life Sciences, University of Chinese Academy of Sciences, Beijing 100049, China
Cheng Tang
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
2College of Life Sciences, University of Chinese Academy of Sciences, Beijing 100049, China
Wenjia Liu
3Department of Ophthalmology, Shanghai General Hospital (Shanghai First People’s Hospital), Shanghai Jiao Tong University School of Medicine, Shanghai, China. Shanghai Key Laboratory of Fundus Diseases, Shanghai, China
Shaoran Wang
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
Yingsi Zhou
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
Qimeng Zhao
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
Qiyu Bo
3Department of Ophthalmology, Shanghai General Hospital (Shanghai First People’s Hospital), Shanghai Jiao Tong University School of Medicine, Shanghai, China. Shanghai Key Laboratory of Fundus Diseases, Shanghai, China
Linyu Shi
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
Xiaodong Sun
3Department of Ophthalmology, Shanghai General Hospital (Shanghai First People’s Hospital), Shanghai Jiao Tong University School of Medicine, Shanghai, China. Shanghai Key Laboratory of Fundus Diseases, Shanghai, China
Haibo Zhou
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
Hui Yang
1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China
Abstract
The smallest Cas13 family protein, CasRx, has a high cleavage activity and targeting specificity, offering attractive opportunity for therapeutic applications. Here we report that delivery of CasRx by adeno-associated virus via intravitreal injection could efficiently knockdown Vegfa transcripts and significantly reduce the area of laser-induced choroidal neovascularization in a mouse model of age-related macular degeneration. Thus, RNA-targeting CRISPR system could be used for in vivo gene therapy.
Copyright
The copyright holder for this preprint is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. All rights reserved. No reuse allowed without permission.
Posted February 20, 2020.
CasRx-mediated RNA targeting prevents choroidal neovascularization in a 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
bioRxiv 2020.02.18.955286; doi: https://doi.org/10.1101/2020.02.18.955286
CasRx-mediated RNA targeting prevents choroidal neovascularization in a 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
bioRxiv 2020.02.18.955286; doi: https://doi.org/10.1101/2020.02.18.955286
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