RNA Interference in model systems of macular degeneration.

渗出性年龄相关性黄斑变性（AMD）是老年人中不可逆的严重视力丧失的最常见原因，全世界估计有2500万至3000万人因AMD而失明。目前还没有针对AMD的标准治疗方法。因此，一种安全、特异、有效的治疗AMD的药物具有巨大的治疗、社会和经济效益。AMD的基础是血管渗漏和新血管形成。我们已经证明，Egr-1，一个核转录因子，在一系列不同的血管生成过程中起着关键的调节作用。在这个项目中，我们将使用新的基因靶向剂（Egr-1 siRNA），以提供其在已建立的AMD动物模型中的治疗潜力的临床前原则证据。这些研究将在AMD患者的IA期安全性试验之前进行。

Exudative age-related macular degeneration (AMD) is the most common cause of irreversible severe vision loss in the elderly, with an estimated 25-30 million people worldwide blind due to AMD. There is currently no standard treatment for AMD. A safe, specific and effective pharmacologic agent for AMD, therefore, has enormous therapeutic, social and economic benefits. AMD is underpinned by vascular leakiness and new blood vessel formation. We have demonstrated that Egr-1, a nuclear transcription factor, plays a key regulatory role in a diverse array of angiogenic processes. In this project, we will use novel gene-targeting agents (Egr-1 siRNA) to provide preclinical proof-of principle evidence of their therapeutic potential in established animal models of AMD. These studies will pre-empt Phase IA safety trials in AMD patients.