Nature Communications (Jul 2021)
A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion
- Jyoti Sharma,
- Ming Du,
- Eric Wong,
- Venkateshwar Mutyam,
- Yao Li,
- Jianguo Chen,
- Jamie Wangen,
- Kari Thrasher,
- Lianwu Fu,
- Ning Peng,
- Liping Tang,
- Kaimao Liu,
- Bini Mathew,
- Robert J. Bostwick,
- Corinne E. Augelli-Szafran,
- Hermann Bihler,
- Feng Liang,
- Jerome Mahiou,
- Josef Saltz,
- Andras Rab,
- Jeong Hong,
- Eric J. Sorscher,
- Eric M. Mendenhall,
- Candice J. Coppola,
- Kim M. Keeling,
- Rachel Green,
- Martin Mense,
- Mark J. Suto,
- Steven M. Rowe,
- David M. Bedwell
Affiliations
- Jyoti Sharma
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Ming Du
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Eric Wong
- CFFT Lab, Cystic Fibrosis Foundation
- Venkateshwar Mutyam
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Yao Li
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Jianguo Chen
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Jamie Wangen
- Department of Molecular Biology and Genetics and Howard Hughes Medical Institute, Johns Hopkins University School of Medicine
- Kari Thrasher
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Lianwu Fu
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Ning Peng
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Liping Tang
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Kaimao Liu
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Bini Mathew
- Southern Research
- Robert J. Bostwick
- Southern Research
- Corinne E. Augelli-Szafran
- Southern Research
- Hermann Bihler
- CFFT Lab, Cystic Fibrosis Foundation
- Feng Liang
- CFFT Lab, Cystic Fibrosis Foundation
- Jerome Mahiou
- CFFT Lab, Cystic Fibrosis Foundation
- Josef Saltz
- CFFT Lab, Cystic Fibrosis Foundation
- Andras Rab
- Department of Pediatrics, Emory University
- Jeong Hong
- Department of Pediatrics, Emory University
- Eric J. Sorscher
- Department of Pediatrics, Emory University
- Eric M. Mendenhall
- Department of Biological Sciences, The University of Alabama in Huntsville
- Candice J. Coppola
- Department of Biological Sciences, The University of Alabama in Huntsville
- Kim M. Keeling
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- Rachel Green
- Department of Molecular Biology and Genetics and Howard Hughes Medical Institute, Johns Hopkins University School of Medicine
- Martin Mense
- CFFT Lab, Cystic Fibrosis Foundation
- Mark J. Suto
- Southern Research
- Steven M. Rowe
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- David M. Bedwell
- Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB)
- DOI
- https://doi.org/10.1038/s41467-021-24575-x
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 16
Abstract
Premature termination codons can cause early translation termination and lead to disease. Here the authors perform a screen to identify compounds with readthrough activity and show that these reduce eRF1 levels to suppress premature termination associated with cystic fibrosis.
