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Article

CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

4 May 2018

Abstract excerpt

LbCpf1, derived from Lachnospiraceae bacterium ND2006, is a CRISPR RNA-guided endonuclease and holds promise for therapeutic applications. Here we show that LbCpf1 can be used for therapeutic gene editing in a mouse model of age-related macular degeneration (AMD). The intravitreal delivery of LbCpf1, targeted to two angiogenesis-associated genes encoding vascular endothelial growth factor A (Vegfa) and hypoxia...

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PMID: 29748595
DOI: 10.1038/s41467-018-04175-y

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