VX-809 and related corrector compounds exhibit secondary activity stabilizing active F508del-CFTR after its partial rescue to the cell surface.

@article{Eckford2014VX809AR,
  title={VX-809 and related corrector compounds exhibit secondary activity stabilizing active F508del-CFTR after its partial rescue to the cell surface.},
  author={Paul D. W. Eckford and Mohabir Ramjeesingh and Steven V Molinski and Stan Pasyk and Johanna F. Dekkers and Canhui Li and Saumel Ahmadi and Wan F Ip and Timothy Ernest Chung and Kai Du and Herman Yeger and Jeffrey Matthijn Beekman and Tanja Gonska and Christine E Bear},
  journal={Chemistry & biology},
  year={2014},
  volume={21 5},
  pages={666-78}
}
The most common mutation causing cystic fibrosis (CF), F508del, impairs conformational maturation of CF transmembrane conductance regulator (CFTR), thereby reducing its functional expression on the surface of epithelia. Corrector compounds including C18 (VRT-534) and VX-809 have been shown to partially rescue misfolding of F508del-CFTR and to enhance its maturation and forward trafficking to the cell surface. Now, we show that there is an additional action conferred by these compounds beyond… CONTINUE READING
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