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EXPERIMENTAL MODELS OF AGE-RELATED MACULAR DEGENERATION AND RELATED RETINAL DISORDERS

机译:年龄相关性黄斑变性和相关性视网膜疾病的实验模型

摘要

The present invention relates to experimental models of age-related macular degeneration (AMD) and related retinal disorders and to screening methods using these models for detecting molecule candidates for the treatment of AMD. In particular, the present invention relates to the use of an animal or a cell that is deficient in functional Niemann Pick type C-related protein as experimental model of AMD and vision disorders resulting from AMD. The present invention founds industrial applications for preclinical research and development on age- related macular degeneration and similar diseases. Examples of potential industrial application are in companies that have a preclinical R&D program in retinal diseases, particularly in AMD.
机译:本发明涉及年龄相关性黄斑变性(AMD)和相关性视网膜疾病的实验模型,以及使用这些模型来检测用于治疗AMD的分子候选物的筛选方法。特别地,本发明涉及缺乏功能性Niemann Pick C型相关蛋白的动物或细胞作为AMD和由AMD引起的视力障碍的实验模型的用途。本发明发现了用于年龄相关性黄斑变性和类似疾病的临床前研究和开发的工业应用。具有潜在临床应用实例的公司包括在视网膜疾病中具有临床前研发计划的公司,尤其是在AMD中。

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