ANTI-ANGIOGENIC GENE THERAPY FOR RETINAL DISEASE

视网膜疾病的抗血管生成基因疗法

• 批准号: 7716020
• 负责人: MARTHA NEURINGER
• 金额: $ 5.55万
• 依托单位: OREGON HEALTH & SCIENCE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-05-01 至 2009-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7716020
• 关键词: Age related macular degeneration; Angiogenesis Inhibitors; Antibodies; Arts; Blindness; Blood Vessels; Computer Retrieval of Information on Scientific Projects Database; Disease Progression; Elderly; Funding; Goals; Grant; Growth; Hemorrhage; Human; Infection; Injection of therapeutic agent; Institution; Lead; Monkeys; Patients; Pongidae; Research; Research Personnel; Resources; Retina; Retinal Detachment; Retinal Diseases; Risk; Source; Testing; United States National Institutes of Health; design; gene therapy; macula; nonhuman primate; research study; therapeutic effectiveness; therapy development

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 is designed to develop a 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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 该项目旨在为年龄相关的黄斑变性（AMD）开发基因疗法，这是老年人中最普遍的失明形式。以最严重的形式，AMD导致脉络膜血管不受控制地侵袭了视网膜，导致出血，视网膜脱落和破坏黄斑视网膜导致中央失明。 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。当前的最新处理涉及每月将抗血管生成抗体注射到玻璃体中。尽管这种治疗方法可有效停止大多数患者的疾病进展，但频繁的眼内注射会带来感染的风险，并可能给某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，该基因疗法可能只需要一次注射即可维持长期的治疗效果。这些研究中非人类灵长类动物的使用至关重要，因为只有猴子和猿具有黄斑和其他类似于人类视网膜的特征。我们的目标是这些实验将导致对人类AMD的有效治疗。