Nature Communications (May 2018)
A novel small molecule chaperone of rod opsin and its potential therapy for retinal degeneration
- Yuanyuan Chen,
- Yu Chen,
- Beata Jastrzebska,
- Marcin Golczak,
- Sahil Gulati,
- Hong Tang,
- William Seibel,
- Xiaoyu Li,
- Hui Jin,
- Yong Han,
- Songqi Gao,
- Jianye Zhang,
- Xujie Liu,
- Hossein Heidari-Torkabadi,
- Phoebe L. Stewart,
- William E. Harte,
- Gregory P. Tochtrop,
- Krzysztof Palczewski
Affiliations
- Yuanyuan Chen
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Yu Chen
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Beata Jastrzebska
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Marcin Golczak
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Sahil Gulati
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Hong Tang
- Drug Discovery Center, University of Cincinnati
- William Seibel
- Drug Discovery Center, University of Cincinnati
- Xiaoyu Li
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Hui Jin
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Yong Han
- Department of Chemistry, Case Western Reserve University
- Songqi Gao
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Jianye Zhang
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Xujie Liu
- Department of Ophthalmology, University of Pittsburgh
- Hossein Heidari-Torkabadi
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- Phoebe L. Stewart
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- William E. Harte
- Office of Translation and Innovation, Case Western Reserve University
- Gregory P. Tochtrop
- Department of Chemistry, Case Western Reserve University
- Krzysztof Palczewski
- Department of Pharmacology, School of Medicine, Case Western Reserve University
- DOI
- https://doi.org/10.1038/s41467-018-04261-1
- Journal volume & issue
-
Vol. 9,
no. 1
pp. 1 – 18
Abstract
Mutations that lead to misfolding of rhodopsin can cause retinitis pigmentosa. Here, the authors carry out a high throughput screen to identify a small molecule chaperone of rod opsin, and show that it protects mouse models of retinitis pigmentosa from retinal degeneration.
