NONHUMAN PRIMATE MODELS OF 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。 视网膜相关性黄斑变性（AMD）是60岁以上成年人视力丧失的主要原因。黄斑是视网膜上的一个特殊区域，是锐利的中央视觉的基础，它只存在于人类和非人类灵长类动物中，因此只有非人类灵长类动物才能为这种复杂的疾病提供准确的模型。其他常见的遗传性视网膜疾病，如色素性视网膜炎和Usher综合征不会选择性地影响黄斑，但在这些疾病中，非人灵长类动物模型对于了解黄斑和中央视觉的保护所涉及的因素（主要治疗目标）也具有独特的价值。AMD的非人灵长类动物模型的可用性将使几种有希望的治疗方法的测试成为可能，包括基因治疗、视网膜移植、生长因子和营养干预，以及视网膜变性的潜在机制的研究。本项目的目标是使用几种互补的方法开发一种非人灵长类动物AMD模型：1）在ONPRC猕猴群中鉴定患有自然发生的黄斑疾病的猴子; 2）确定这种疾病背后的基因突变或易感因素和基因表达变化; 3）测试视网膜基因治疗的可行性，安全性和有效性;和4）测试长期的、控制的饮食干预的效果，其可以保护黄斑免于黄斑变性。

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. Age-related macular degeneration (AMD) is the leading cause of vision loss in adults over 60 years of age. The macula, the specialized area of the retina that underlies sharp central vision, is present only in human and nonhuman primates, so that only nonhuman primates can provide an accurate model for this complex disease. Other common inherited retinal diseases such as retinitis pigmentosa and Usher syndrome do not selectively affect the macula, but in these diseases also, a nonhuman primate model would be uniquely valuable for understanding the factors involved in preservation of the macula and central vision, a primary therapeutic goal. Availability of a nonhuman primate model of AMD would make possible tests of several promising therapeutic approaches, including gene therapy, retinal transplantation, growth factors and nutritional interventions, as well as studies of the mechanisms underlying retinal degeneration. This project's objective is to develop a nonhuman primate model for AMD using several complementary approaches: 1) identifying monkeys with naturally-occurring macular disease in the ONPRC macaque colonies; 2) determining the genetic mutations or susceptibility factors and gene expression changes underlying this disease; 3) testing the feasibility, safety and efficacy of retinal gene therapies; and 4) testing the effects of long-term, controlled dietary interventions that may protect the macula from macular degeneration.