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A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion.
Sharma J, Du M, Wong E, Mutyam V, Li Y, Chen J, Wangen J, Thrasher K, Fu L, Peng N, Tang L, Liu K, Mathew B, Bostwick RJ, Augelli-Szafran CE, Bihler H, Liang F, Mahiou J, Saltz J, Rab A, Hong J, Sorscher EJ, Mendenhall EM, Coppola CJ, Keeling KM, Green R, Mense M, Suto MJ, Rowe SM, Bedwell DM. Sharma J, et al. Among authors: chen j. Nat Commun. 2021 Jul 16;12(1):4358. doi: 10.1038/s41467-021-24575-x. Nat Commun. 2021. PMID: 34272367 Free PMC article.
The synthetic aminoglycoside ELX-02 induces readthrough of G550X-CFTR producing superfunctional protein that can be further enhanced by CFTR modulators.
Chen J, Thrasher K, Fu L, Wang W, Aghamohammadzadeh S, Wen H, Tang L, Keeling KM, Falk Libby E, Bedwell DM, Rowe SM. Chen J, et al. Am J Physiol Lung Cell Mol Physiol. 2023 Jun 1;324(6):L756-L770. doi: 10.1152/ajplung.00038.2023. Epub 2023 Apr 4. Am J Physiol Lung Cell Mol Physiol. 2023. PMID: 37014818 Free PMC article.
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