Article
A novel small molecule chaperone of rod opsin and its potential therapy for retinal degeneration.
Nature communications - 17 May 2018
Chen Yuanyuan, Chen Yu, Jastrzebska Beata, Golczak Marcin, Gulati Sahil, Tang Hong, Seibel William, Li Xiaoyu, Jin Hui, Han Yong, Gao Songqi, Zhang Jianye, Liu Xujie, Heidari-Torkabadi Hossein, Stewart Phoebe L, Harte William E, Tochtrop Gregory P, Palczewski Krzysztof
Abstract excerpt
Rhodopsin homeostasis is tightly coupled to rod photoreceptor cell survival and vision. Mutations resulting in the misfolding of rhodopsin can lead to autosomal dominant retinitis pigmentosa (adRP), a progressive retinal degeneration that currently is untreatable. Using a cell-based high-throughput screen (HTS) to identify small molecules that can stabilize the P23H-opsin mutant, which causes most cases of adRP,...
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