‘Hit and run’ therapy averts macular degeneration

NATURE BIOMEDICAL ENGINEERING（2021）

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摘要

Subretinal delivery of lentiviruses bearing Cas9 mRNA and a guide RNA targeting the Vegfa gene reduces the development of choroidal neovascularization in a mouse model of wet age-related macular degeneration.

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关键词

Expression systems,Gene therapy,Macular degeneration,Biomedicine,general,Biomedical Engineering/Biotechnology

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