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AAV-CRISPR/Cas9 Genome Editing of VEGFR2 for Treating Ocular Diseases
专利权人:
Massachusetts Eye and Ear Infirmary
发明人:
Hetian Lei
申请号:
US16621889
公开号:
US20200113926A1
申请日:
2018.06.27
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Editing of VEGFR2 abrogated angiogenesis in two mouse models of oxygen-induced retinopathy (OIR) and laser-induced choroid neovascularization (CNV). Provided are compositions, e.g., Adeno-Associated Virus (AAV) Vectors comprising sequences encoding CRISPR/Cas9 proteins and guide RNA, and methods of use thereof for editing of Vascular endothelial growth factor receptor 2 (VEGFR2) gene to treat ocular disease associated with pathological angiogenesis, e.g., neovascular age-related macular degeneration (AMD), proliferative diabetic retinopathy (PDR) and retinopathy of prematurity (ROP).
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