申请人:CASE WESTERN RESERVE UNIVERSITY
公开号:US11191752B2
公开(公告)日:2021-12-07
This application relates to compounds and methods of treating retinal degeneration associated with inherited rhodopsin mutations in the ocular tissue of a subject. The retinal degeneration, can include, for example, macular degeneration, a including age-related macular degeneration, Stargardt disease, and retinitis pigmentosa. The retinitis pigmentosa can include autosomal dominate retinitis pigmentosa associated with a P23H RHO mutation. A method of treating retinal degeneration in a subject includes administering to the subject a therapeutically effective amount of a compound of formula (I), wherein the compound of formula (I) acts as a chaperone of rhodopsin.
本申请涉及治疗与受试者眼组织中遗传性视网膜视蛋白突变有关的视网膜变性的化合物和方法。视网膜变性可包括例如黄斑变性,包括老年性黄斑变性、Stargardt 病和视网膜色素变性。视网膜色素变性可包括与 P23H RHO 突变相关的常染色体显性视网膜色素变性。一种治疗受试者视网膜变性的方法包括向受试者施用治疗有效量的式(I)化合物,其中式(I)化合物作为视紫红质的伴侣蛋白。