Experimental models of age-related macular degeneration and related retinal disorders

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.