Pechan Peter, Wadsworth Samuel, Scaria Abraham
Gene Therapy, Sanofi-Genzyme R&D Center, Framingham, Massachusetts 01701.
Cold Spring Harb Perspect Med. 2014 Dec 18;5(7):a017335. doi: 10.1101/cshperspect.a017335.
Pathological neovascularization is a key component of the neovascular form (also known as the wet form) of age-related macular degeneration (AMD) and proliferative diabetic retinopathy. Several preclinical studies have shown that antiangiogenesis strategies are effective for treating neovascular AMD in animal models. Vascular endothelial growth factor (VEGF) is one of the main inducers of ocular neovascularization, and several clinical trials have shown the benefits of neutralizing VEGF in patients with neovascular AMD or diabetic macular edema. In this review, we summarize several preclinical and early-stage clinical trials with intraocular gene therapies, which have the potential to reduce or eliminate the repeated intravitreal injections that are currently required for the treatment of neovascular AMD.
病理性血管生成是年龄相关性黄斑变性(AMD)的新生血管型(也称为湿性型)和增殖性糖尿病视网膜病变的关键组成部分。多项临床前研究表明,抗血管生成策略在动物模型中对治疗新生血管性AMD有效。血管内皮生长因子(VEGF)是眼部血管生成的主要诱导因子之一,多项临床试验已证明中和VEGF对新生血管性AMD或糖尿病性黄斑水肿患者有益。在本综述中,我们总结了几项眼内基因治疗的临床前和早期临床试验,这些试验有可能减少或消除目前治疗新生血管性AMD所需的重复玻璃体内注射。