CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration

Author:

Zhou Changyang12,Hu Xinde12,Tang Cheng12,Liu Wenjia3,Wang Shaoran14,Zhou Yingsi1,Zhao Qimeng1,Bo Qiyu3,Shi Linyu14,Sun Xiaodong3,Zhou Haibo1,Yang Hui1

Affiliation:

1. Institute 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, China

2. College of Life Sciences, University of Chinese Academy of Sciences, China

3. Department of Ophthalmology, Shanghai General Hospital (Shanghai First People's Hospital), Shanghai Jiao Tong University School of Medicine, Shanghai Key Laboratory of Fundus Diseases, China

4. Hui-Gene Therapeutics Co., Ltd. China

Abstract

Summary RNA-targeting CRISPR system Cas13 offers an efficient approach for manipulating RNA transcripts in vitro. In this perspective, we provide a proof-of-concept demonstration that Cas13-mediated Vegfa knockdown in vivo could prevent the development of laser-induced CNV in mouse model of Age-related macular degeneration.

Funder

R&D Program of China

Strategic Priority Research Program

National Natural Science Foundation of China

Shanghai Municipal Science and Technology

Shanghai City Committee of science and technology

Publisher

Oxford University Press (OUP)

Subject

Multidisciplinary

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