Genome editing abrogates angiogenesis in vivo

Xionggao Huang, Guohong Zhou, Wenyi Wu, Yajian Duan, Gaoen Ma, Jingyuan Song, Ru Xiao, Luk Vandenberghe, Feng Zhang, Patricia A. D’Amore and Hetian Lei ()
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Xionggao Huang: Harvard Medical School
Guohong Zhou: Harvard Medical School
Wenyi Wu: Harvard Medical School
Yajian Duan: Harvard Medical School
Gaoen Ma: Harvard Medical School
Jingyuan Song: Harvard Medical School
Ru Xiao: Harvard Medical School
Luk Vandenberghe: Harvard Medical School
Feng Zhang: Broad Institute of the Massachusetts Institute of Technology and Harvard University
Patricia A. D’Amore: Harvard Medical School
Hetian Lei: Harvard Medical School

Nature Communications, 2017, vol. 8, issue 1, 1-8

Abstract: Abstract Angiogenesis, in which vascular endothelial growth factor receptor (VEGFR) 2 plays an essential role, is associated with a variety of human diseases including proliferative diabetic retinopathy and wet age-related macular degeneration. Here we report that a system of adeno-associated virus (AAV)-mediated clustered regularly interspaced short palindromic repeats (CRISPR)-associated endonuclease (Cas)9 from Streptococcus pyogenes (SpCas9) is used to deplete VEGFR2 in vascular endothelial cells (ECs), whereby the expression of SpCas9 is driven by an endothelial-specific promoter of intercellular adhesion molecule 2. We further show that recombinant AAV serotype 1 (rAAV1) transduces ECs of pathologic vessels, and that editing of genomic VEGFR2 locus using rAAV1-mediated CRISPR/Cas9 abrogates angiogenesis in the mouse models of oxygen-induced retinopathy and laser-induced choroid neovascularization. This work establishes a strong foundation for genome editing as a strategy to treat angiogenesis-associated diseases.

Date: 2017
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DOI: 10.1038/s41467-017-00140-3

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