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

DOI
https://doi.org/10.1038/s41467-021-24575-x
Journal volume & issue
Vol. 12, no. 1
pp. 1 – 16

Abstract

Read online

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.