CASE WESTERN RESERVE UNIVERSITY;UNIVERSITY OF PITTSBURGH
发明人:
Krzysztof Palczewski,Yuanyuan Chen
申请号：
US16609162
公开号：
US20200230119A1
申请日:
2018.04.30
申请国别(地区):
US
年份:
2020
代理人:
摘要：
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.
