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유전체교정 연구단
유전체 교정 연구단
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CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

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Title
CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration
Author(s)
Taeyoung Koo; Sung Wook Park; Dong Hyun Jo; Daesik Kim; Jin Hyoung Kim; Hee-Yeon Cho; Jeungeun Kim; jeong Hun Kim; Jin-Soo Kim
Publication Date
2018-05
Journal
NATURE COMMUNICATIONS, v.9, no.1, pp.1855 -
Publisher
NATURE PUBLISHING GROUP
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), using adeno-associated virus, led to efficient gene disruption with no apparent off-target effects in the retina and retinal pigment epithelium (RPE) cells. Importantly, LbCpf1 targeted to Vegfa or Hif1a in RPE cells reduced the area of laser-induced choroidal neovascularization as efficiently as aflibercept, an anti-VEGF drug currently used in the clinic, without inducing cone dysfunction. Unlike aflibercept, LbCpf1 targeted to Vegfa or Hif1a achieved a long-term therapeutic effect on CNV, potentially avoiding repetitive injections. Taken together, these results indicate that LbCpf1-mediated in vivo genome editing to ablate pathologic angiogenesis provides an effective strategy for the treatment of AMD and other neovascularization-associated diseases. © 2018 The Author(s)
URI
https://pr.ibs.re.kr/handle/8788114/4513
ISSN
2041-1723
Appears in Collections:
Center for Genome Engineering(유전체 교정 연구단) > Journal Papers (저널논문)
Files in This Item:
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