Author: Zhou, Changyang; Hu, Xinde; Tang, Cheng; Liu, Wenjia; Wang, Shaoran; Zhou, Yingsi; Zhao, Qimeng; Bo, Qiyu; Shi, Linyu; Sun, Xiaodong; Zhou, Haibo; Yang, Hui
Title: CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration Cord-id: deu7lrxq Document date: 2020_2_20
ID: deu7lrxq
Snippet: 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 ther
Document: 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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