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

Volume: 9, Issue: 1
Published: May 10, 2018
Abstract
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 inducing factor 1a ( Hif1a ),...
Paper Details
Title
CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration
Published Date
May 10, 2018
Volume
9
Issue
1
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