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正常角膜是无血管、完全透明的组织,是眼部重要的屈光介质,但许多眼部疾病均可破坏抗血管生成因子与促血管生成因子之间的平衡,导致病理性角膜新生血管(CNV)的形成.大量研究表明,CNV的形成与血管内皮生长因子(VEGF)信号通路的激活密切相关.通过多靶点多途径阻断该信号通路可以有效抑制新生血管的形成,为CNV的治疗带来了希望.目前,针对新生血管性眼病的新型靶向治疗策略主要包括VEGF抑制剂和以微小RNA(miRNA)为核心的基因治疗,前者主要包括抗VEGF单克隆抗体、核酸适体、VEGFtrap、VEGF受体(VEGFR)酪氨酸激酶抑制剂等.本文将对具有代表性的抗VEGF药物和基因治疗的作用机制、用药疗效、药物安全性及研究现状进行综述.“,”Normal cornea is avascular,fully transparent organization,which is important to the eye refraction.However,many eye diseases can destroy the anti-angiogenic factor and pro-angiogenic factors balance,resulting in pathological corneal neovascularization (CNV) formation.Numerous studies show that activation of signaling pathways are closely related to CNV formation and vascular endothelial growth factor (VEGF).Multiple targets and multichannel blocker can effectively inhibit the signaling pathway of angiogenesis,which brought hope for the treatment of CNV.Currently new targeted treatment strategy to ocular neovascularization include VEGF inhibitors and with the core of miRNA gene therapy.The former mainly include anti-VEGF monoclonal antibodies,aptamers,VEGF trap,VEGF receptor (VEGFR) tyrosine kinase inhibitor,etc.This article reviewed the mechanism of action,drug efficacy,drug safety and research status of representative anti-VEGF drugs and Gene therapy.