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Revertants, low temperature, and correctors reveal the mechanism of F508del-CFTR rescue by VX-809 and suggest multiple agents for full correction.

Farinha CM, King-Underwood J, Sousa M, Correia AR, Henriques BJ, Roxo-Rosa M, Da Paula AC, Williams J, Hirst S, Gomes CM, Amaral MD.

Chem Biol. 2013 Jul 25;20(7):943-55. doi: 10.1016/j.chembiol.2013.06.004.


Searching for combinations of small-molecule correctors to restore f508del-cystic fibrosis transmembrane conductance regulator function and processing.

Boinot C, Jollivet Souchet M, Ferru-Clément R, Becq F.

J Pharmacol Exp Ther. 2014 Sep;350(3):624-34. doi: 10.1124/jpet.114.214890. Epub 2014 Jun 26.


VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1.

Ren HY, Grove DE, De La Rosa O, Houck SA, Sopha P, Van Goor F, Hoffman BJ, Cyr DM.

Mol Biol Cell. 2013 Oct;24(19):3016-24. doi: 10.1091/mbc.E13-05-0240. Epub 2013 Aug 7.


Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770.

Gentzsch M, Ren HY, Houck SA, Quinney NL, Cholon DM, Sopha P, Chaudhry IG, Das J, Dokholyan NV, Randell SH, Cyr DM.

Am J Physiol Lung Cell Mol Physiol. 2016 Sep 1;311(3):L550-9. doi: 10.1152/ajplung.00186.2016. Epub 2016 Jul 8.


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

Phuan PW, Veit G, Tan J, Roldan A, Finkbeiner WE, Lukacs GL, Verkman AS.

Mol Pharmacol. 2014 Jul;86(1):42-51. doi: 10.1124/mol.114.092478. Epub 2014 Apr 15.


Capturing the Direct Binding of CFTR Correctors to CFTR by Using Click Chemistry.

Sinha C, Zhang W, Moon CS, Actis M, Yarlagadda S, Arora K, Woodroofe K, Clancy JP, Lin S, Ziady AG, Frizzell R, Fujii N, Naren AP.

Chembiochem. 2015 Sep 21;16(14):2017-22. doi: 10.1002/cbic.201500123. Epub 2015 Aug 11.


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

Eckford PD, Ramjeesingh M, Molinski S, Pasyk S, Dekkers JF, Li C, Ahmadi S, Ip W, Chung TE, Du K, Yeger H, Beekman J, Gonska T, Bear CE.

Chem Biol. 2014 May 22;21(5):666-78. doi: 10.1016/j.chembiol.2014.02.021. Epub 2014 Apr 10.


Probing conformational rescue induced by a chemical corrector of F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutant.

Yu W, Kim Chiaw P, Bear CE.

J Biol Chem. 2011 Jul 15;286(28):24714-25. doi: 10.1074/jbc.M111.239699. Epub 2011 May 21.


Mechanism-based corrector combination restores ΔF508-CFTR folding and function.

Okiyoneda T, Veit G, Dekkers JF, Bagdany M, Soya N, Xu H, Roldan A, Verkman AS, Kurth M, Simon A, Hegedus T, Beekman JM, Lukacs GL.

Nat Chem Biol. 2013 Jul;9(7):444-54. doi: 10.1038/nchembio.1253. Epub 2013 May 12.


Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.

Van Goor F, Hadida S, Grootenhuis PD, Burton B, Stack JH, Straley KS, Decker CJ, Miller M, McCartney J, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu PA.

Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18843-8. doi: 10.1073/pnas.1105787108. Epub 2011 Oct 5.


A molecular switch in the scaffold NHERF1 enables misfolded CFTR to evade the peripheral quality control checkpoint.

Loureiro CA, Matos AM, Dias-Alves Â, Pereira JF, Uliyakina I, Barros P, Amaral MD, Matos P.

Sci Signal. 2015 May 19;8(377):ra48. doi: 10.1126/scisignal.aaa1580.


F508del-cystic fibrosis transmembrane regulator correctors for treatment of cystic fibrosis: a patent review.

Yang H, Ma T.

Expert Opin Ther Pat. 2015;25(9):991-1002. doi: 10.1517/13543776.2015.1045878. Epub 2015 May 15. Review.


Identification of a NBD1-binding pharmacological chaperone that corrects the trafficking defect of F508del-CFTR.

Sampson HM, Robert R, Liao J, Matthes E, Carlile GW, Hanrahan JW, Thomas DY.

Chem Biol. 2011 Feb 25;18(2):231-42. doi: 10.1016/j.chembiol.2010.11.016.


Increasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule Therapeutics.

Chung WJ, Goeckeler-Fried JL, Havasi V, Chiang A, Rowe SM, Plyler ZE, Hong JS, Mazur M, Piazza GA, Keeton AB, White EL, Rasmussen L, Weissman AM, Denny RA, Brodsky JL, Sorscher EJ.

PLoS One. 2016 Oct 12;11(10):e0163615. doi: 10.1371/journal.pone.0163615. eCollection 2016.


Integrated biophysical studies implicate partial unfolding of NBD1 of CFTR in the molecular pathogenesis of F508del cystic fibrosis.

Wang C, Protasevich I, Yang Z, Seehausen D, Skalak T, Zhao X, Atwell S, Spencer Emtage J, Wetmore DR, Brouillette CG, Hunt JF.

Protein Sci. 2010 Oct;19(10):1932-47. doi: 10.1002/pro.480.


Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis.

Awatade NT, Uliyakina I, Farinha CM, Clarke LA, Mendes K, Solé A, Pastor J, Ramos MM, Amaral MD.

EBioMedicine. 2014 Dec 17;2(2):147-53. doi: 10.1016/j.ebiom.2014.12.005. eCollection 2015.


The major cystic fibrosis causing mutation exhibits defective propensity for phosphorylation.

Pasyk S, Molinski S, Ahmadi S, Ramjeesingh M, Huan LJ, Chin S, Du K, Yeger H, Taylor P, Moran MF, Bear CE.

Proteomics. 2015 Jan;15(2-3):447-61. doi: 10.1002/pmic.201400218. Epub 2014 Dec 17.


Corrector VX-809 stabilizes the first transmembrane domain of CFTR.

Loo TW, Bartlett MC, Clarke DM.

Biochem Pharmacol. 2013 Sep 1;86(5):612-9. doi: 10.1016/j.bcp.2013.06.028. Epub 2013 Jul 5.


Decoding F508del misfolding in cystic fibrosis.

Wang XR, Li C.

Biomolecules. 2014 May 6;4(2):498-509. doi: 10.3390/biom4020498. Review.


The investigational Cystic Fibrosis drug Trimethylangelicin directly modulates CFTR by stabilizing the first membrane-spanning domain.

Laselva O, Molinski S, Casavola V, Bear CE.

Biochem Pharmacol. 2016 Nov 1;119:85-92. doi: 10.1016/j.bcp.2016.09.005. Epub 2016 Sep 8.


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