Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.

@article{Phuan2014SynergybasedSS,
  title={Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.},
  author={Puay-Wah Phuan and Guido Veit and Joseph A Tan and Ariel Rold{\'a}n and Walter E. Finkbeiner and Gergely L Lukacs and Alan S. Verkman},
  journal={Molecular pharmacology},
  year={2014},
  volume={86 1},
  pages={42-51}
}
The most prevalent cystic fibrosis transmembrane conductance regulator (CFTR) mutation causing cystic fibrosis, ΔF508, impairs folding of nucleotide binding domain (NBD) 1 and stability of the interface between NBD1 and the membrane-spanning domains. The interfacial stability defect can be partially corrected by the investigational drug VX-809 (3-[6-[[[1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropyl]carbonyl]amino]-3-methyl-2-pyridinyl]-benzoic acid) or the R1070W mutation. Second-generation… CONTINUE READING

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Correction of both NBD1

  • WM Rabeh, F Bossard, +7 authors L Konermann
  • 2012
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