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CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration
[article]
2020
bioRxiv
pre-print
Preserved Fulltext
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.
doi:10.1101/2020.02.18.955286
fatcat:ugecwwydivbixjjcgyk7h4ln7q