Pegaptanib sodium for ocular vascular disease.
Pegaptanib sodium for ocular vascular disease.
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DOI：
10.4103/0301-4738.36476
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发表时间：
2007-11
影响因子：
3.1
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中科院分区：
文献类型：
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哌加他尼钠（MacugenTM）是一种选择性RNA适体，其抑制血管内皮生长因子（VEGF）165，VEGF同种型主要负责病理性眼部新血管形成和血管渗透性，同时保留生理性同种型VEGF 121。经过10多年的开发和临床前研究，临床试验显示哌加他尼可有效治疗与年龄相关性黄斑变性相关的脉络膜新生血管。其出色的眼部和全身安全性也已在接受长达三年治疗的患者中得到证实。早期对照良好的研究进一步表明，哌加他尼可能为糖尿病黄斑水肿、增殖性糖尿病视网膜病变和视网膜静脉阻塞患者提供治疗获益。值得注意的是，哌加他尼是第一个被批准用于人类治疗的适体，也是第一个可用于治疗眼血管疾病的VEGF抑制剂。
Pegaptanib sodium (MacugenTM) is a selective RNA aptamer that inhibits vascular endothelial growth factor (VEGF) 165 , the VEGF isoform primarily responsible for pathologic ocular neovascularization and vascular permeability, while sparing the physiological isoform VEGF 121 . After more than 10 years in development and preclinical study, pegaptanib was shown in clinical trials to be effective in treating choroidal neovascularization associated with age-related macular degeneration. Its excellent ocular and systemic safety profile has also been confirmed in patients receiving up to three years of therapy. Early, well-controlled studies further suggest that pegaptanib may provide therapeutic benefit for patients with diabetic macular edema, proliferative diabetic retinopathy and retinal vein occlusion. Notably, pegaptanib was the first available aptamer approved for therapeutic use in humans and the first VEGF inhibitor available for the treatment of ocular vascular diseases.