Nature Communications (May 2018)
CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration
Abstract
The CRISPR endonuclease LbCpf1 is reported to have greater efficiency and specificity than Cas9. Here, the authors use LbCpf1 to target the angiogenesis-related genes VEGF and HIF1a, and show that delivery of the nuclease using AAV9 is effective in mouse models of macular degeneration.