ANTI-ANGIOGENIC GENE THERAPY FOR RETINAL DISEASE

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 该项目旨在测试一种潜在的老年性黄斑变性（AMD）基因疗法，AMD是老年人最常见的失明形式。在其最严重的形式中，AMD导致脉络膜血管对视网膜的不受控制的侵入，导致出血、视网膜脱离和黄斑视网膜的破坏，引起中心性失明。治疗开发的努力主要集中在用抗血管生成剂阻止血管生长。目前最先进的治疗方法包括每月向玻璃体注射抗血管生成抗体。虽然这种治疗在阻止大多数患者的疾病进展方面是有效的，但频繁的眼内注射会带来感染的风险，并可能给一些患者带来不可接受的负担。我们正在测试一种潜在的基因疗法，可能只需要一次注射就可以保持长期的治疗效果。在这些研究中使用非人类灵长类动物是至关重要的，因为只有猴子和猿类具有与人类视网膜非常相似的黄斑和其他特征。我们的目标是这些实验将导致人类AMD的有效治疗。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This project was designed to test a potential genetic therapy for age-related macular degeneration (AMD), the most prevalent form of blindness in the elderly. In its most severe form AMD results in uncontrolled invasion of the retina by choroidal blood vessels leading to hemorrhage, retinal detachment and destruction of the macular retina causing central blindness. Efforts at therapy development have largely focused on stopping vessel growth with anti-angiogenic agents. The current state-of-the-art treatment involves monthly injection of anti-angiogenic antibodies into the vitreous. Although this treatment is effective in stopping disease progression in a majority of patients, frequent intraocular injections pose risk of infection and can present an unacceptable burden to some patients. We are testing a potential gene therapy that could require only one injection to maintain long-term therapeutic effectiveness. The use of nonhuman primates for these studies is critical because only monkeys and apes have a macula and other features closely resembling the human retina. It is our goal that these experiments will lead to an effective treatment for AMD in humans.