National Science ReviewGeneticsPeer-reviewed journal article
CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration
Changyang ZhouiD, Xinde HuiD, Cheng TangiD, Wenjia LiuiD, Shaoran WangiD, Yingsi ZhouiD, Qimeng Zhao, Qiyu BoiD, Linyu ShiiD, Xiaodong SuniD, Haibo ZhouiD, Hui YangiD
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.
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