Article
Pharmacological clearance of misfolded rhodopsin for the treatment of RHO-associated retinitis pigmentosa.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology - 1 Aug 2020
Liu Xujie, Feng Bing, Vats Abhishek, Tang Hong, Seibel William, Swaroop Manju, Tawa Gregory, Zheng Wei, Byrne Leah, Schurdak Mark, Chen Yuanyuan
Abstract excerpt
Rhodopsin mutation and misfolding is a common cause of autosomal dominant retinitis pigmentosa (RP). Using a luciferase reporter assay, we undertook a small-molecule high-throughput screening (HTS) of 68, 979 compounds and identified nine compounds that selectively reduced the misfolded P23H rhodopsin without an effect on the wild type (WT) rhodopsin protein. Further, we found five of these compounds, including...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
