【摘 要】
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Age-related macular degeneration (AMD),characterized by the development of choroidal neovascularization (CNV),is a leading cause of vision deterioration in adul
【机 构】
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Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiol
论文部分内容阅读
Age-related macular degeneration (AMD),characterized by the development of choroidal neovascularization (CNV),is a leading cause of vision deterioration in adults over the age of 50 [1].An angiogenic growth factor vascular endothelial growth factor A (VEGFA)plays a crucial role in CNV pathogenesis and anti-VEGFA therapy using humanized antibodies has been widely used in treating AMD,with the therapeutic effects maintained by regular injections of antibodies [2,3].Two recent studies showed in a mouse model of AMD that permanent Vegfa gene disruption could be induced by spCas9 or LbCpf1 editing [4,5].However,risks associated with permanent DNA modifications,including unwanted off-target and on-target effects,need to be considered [6,7].
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