-
1
-
-
84890435909
-
Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function
-
Van Goor F, Yu H, Burton B, et al. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros 2014; 13: 29-36.
-
(2014)
J Cyst Fibros
, vol.13
, pp. 29-36
-
-
Van Goor, F.1
Yu, H.2
Burton, B.3
-
2
-
-
84862776940
-
Ivacaftor potentiation of multiple CFTR channels with gating mutations
-
Yu H, Burton B, Huang CJ, et al. Ivacaftor potentiation of multiple CFTR channels with gating mutations. J Cyst Fibros 2012; 11: 237-245.
-
(2012)
J Cyst Fibros
, vol.11
, pp. 237-245
-
-
Yu, H.1
Burton, B.2
Huang, C.J.3
-
3
-
-
0025242929
-
Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis
-
Cheng SH, Gregory RJ, Marshall J, et al. Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis. Cell 1990; 63: 827-834.
-
(1990)
Cell
, vol.63
, pp. 827-834
-
-
Cheng, S.H.1
Gregory, R.J.2
Marshall, J.3
-
4
-
-
84904002908
-
A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: A phase 2 randomised controlled trial
-
Boyle MP, Bell SC, Konstan MW, et al. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med 2014; 2: 527-538.
-
(2014)
Lancet Respir Med
, vol.2
, pp. 527-538
-
-
Boyle, M.P.1
Bell, S.C.2
Konstan, M.W.3
-
5
-
-
84937035647
-
Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR
-
Wainwright CE, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015; 373: 220-231.
-
(2015)
N Engl J Med
, vol.373
, pp. 220-231
-
-
Wainwright, C.E.1
-
6
-
-
24644464284
-
Small-molecule correctors of defective Δf508-CFTR cellular processing identified by high-throughput screening
-
Pedemonte N, Lukacs GL, Du K, et al. Small-molecule correctors of defective ΔF508-CFTR cellular processing identified by high-throughput screening. J Clin Invest 2005; 115: 2564-2571.
-
(2005)
J Clin Invest
, vol.115
, pp. 2564-2571
-
-
Pedemonte, N.1
Lukacs, G.L.2
Du, K.3
-
7
-
-
33744831154
-
Rescue of Δf508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules
-
Van Goor F, Straley KS, Cao D, et al. Rescue of ΔF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol Lung Cell Mol Physiol 2006; 290: L1117-L1130.
-
(2006)
Am J Physiol Lung Cell Mol Physiol
, vol.290
, pp. L1117-L1130
-
-
Van Goor, F.1
Straley, K.S.2
Cao, D.3
-
8
-
-
33645530653
-
The chemical chaperone CFcor-325 repairs folding defects in the transmembrane domains of CFTR-processing mutants
-
Loo TW, Bartlett MC, Wang Y, et al. The chemical chaperone CFcor-325 repairs folding defects in the transmembrane domains of CFTR-processing mutants. Biochem J 2006; 395: 537-542.
-
(2006)
Biochem J
, vol.395
, pp. 537-542
-
-
Loo, T.W.1
Bartlett, M.C.2
Wang, Y.3
-
9
-
-
15944387059
-
Discovery of 1,2,3,4-tetrahydroisoquinoline-3-carboxylic acid diamides that increase CFTR mediated chloride transport
-
Hirth BH, Qiao S, Cuff LM, et al. Discovery of 1,2,3,4-tetrahydroisoquinoline-3-carboxylic acid diamides that increase CFTR mediated chloride transport. Bioorg Med Chem Lett 2005; 15: 2087-2091.
-
(2005)
Bioorg Med Chem Lett
, vol.15
, pp. 2087-2091
-
-
Hirth, B.H.1
Qiao, S.2
Cuff, L.M.3
-
10
-
-
38549125726
-
Structural analog of sildenafil identified as a novel corrector of the F508del-CFTR trafficking defect
-
Robert R, Carlile GW, Pavel C, et al. Structural analog of sildenafil identified as a novel corrector of the F508del-CFTR trafficking defect. Mol Pharmacol 2008; 73: 478-489.
-
(2008)
Mol Pharmacol
, vol.73
, pp. 478-489
-
-
Robert, R.1
Carlile, G.W.2
Pavel, C.3
-
11
-
-
33646892646
-
Dynasore, a cell-permeable inhibitor of dynamin
-
Macia E, Ehrlich M, Massol R, et al. Dynasore, a cell-permeable inhibitor of dynamin. Dev Cell 2006; 10: 839-850.
-
(2006)
Dev Cell
, vol.10
, pp. 839-850
-
-
Macia, E.1
Ehrlich, M.2
Massol, R.3
-
12
-
-
41849133213
-
4'-Methyl-4,5'-bithiazole-based correctors of defective Δf508-CFTR cellular processing
-
Yoo CL, Yu GJ, Yang B, et al. 4'-Methyl-4,5'-bithiazole-based correctors of defective ΔF508-CFTR cellular processing. Bioorg Med Chem Lett 2008; 18: 2610-2614.
-
(2008)
Bioorg Med Chem Lett
, vol.18
, pp. 2610-2614
-
-
Yoo, C.L.1
Yu, G.J.2
Yang, B.3
-
13
-
-
78751644734
-
Paneth cells constitute the niche for Lgr5 stem cells in intestinal crypts
-
Sato T, van Es JH, Snippert HJ, et al. Paneth cells constitute the niche for Lgr5 stem cells in intestinal crypts. Nature 2011; 469: 415-418.
-
(2011)
Nature
, vol.469
, pp. 415-418
-
-
Sato, T.1
Van Es, J.H.2
Snippert, H.J.3
-
14
-
-
80054857419
-
Long-term expansion of epithelial organoids from human colon, adenoma, adenocarcinoma, and Barrett's epithelium
-
Sato T, Stange DE, Ferrante M, et al. Long-term expansion of epithelial organoids from human colon, adenoma, adenocarcinoma, and Barrett's epithelium. Gastroenterology 2011; 141: 1762-1772.
-
(2011)
Gastroenterology
, vol.141
, pp. 1762-1772
-
-
Sato, T.1
Stange, D.E.2
Ferrante, M.3
-
15
-
-
80053978141
-
Isolation and in vitro expansion of human colonic stem cells
-
Jung P, et al. Isolation and in vitro expansion of human colonic stem cells. Nat Med 2011; 17: 1225-1227.
-
(2011)
Nat Med
, vol.17
, pp. 1225-1227
-
-
Jung, P.1
-
16
-
-
84880292828
-
A functional CFTR assay using primary cystic fibrosis intestinal organoids
-
Dekkers JF, Wiegerinck CL, de Jonge HR, et al. A functional CFTR assay using primary cystic fibrosis intestinal organoids. Nat Med 2013; 19: 939-945.
-
(2013)
Nat Med
, vol.19
, pp. 939-945
-
-
Dekkers, J.F.1
Wiegerinck, C.L.2
De Jonge, H.R.3
-
17
-
-
84902335825
-
Novel opportunities for CFTR-targeting drug development using organoids
-
Dekkers JF, van der Ent CK, Beekman JM. Novel opportunities for CFTR-targeting drug development using organoids. Rare Dis 2013; 1: e27112.
-
(2013)
Rare Dis
, vol.1
, pp. e27112
-
-
Dekkers, J.F.1
Van Der Ent, C.K.2
Beekman, J.M.3
-
18
-
-
84896913799
-
Correcting the cystic fibrosis disease mutant, A455E CFTR
-
Cebotaru L, Rapino D, Cebotaru V, et al. Correcting the cystic fibrosis disease mutant, A455E CFTR. PLoS One 2014; 9: e85183.
-
(2014)
PLoS One
, vol.9
, pp. e85183
-
-
Cebotaru, L.1
Rapino, D.2
Cebotaru, V.3
-
19
-
-
84879410121
-
Mechanism-based corrector combination restores Δf508-CFTR folding and function
-
Okiyoneda T, Veit G, Dekkers JF, et al. Mechanism-based corrector combination restores ΔF508-CFTR folding and function. Nat Chem Biol 2013; 9: 444-454.
-
(2013)
Nat Chem Biol
, vol.9
, pp. 444-454
-
-
Okiyoneda, T.1
Veit, G.2
Dekkers, J.F.3
-
20
-
-
84890435909
-
Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function
-
Van Goor F, Yu H, Burton B, et al. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros 2014; 13: 29-36.
-
(2014)
J Cyst Fibros
, vol.13
, pp. 29-36
-
-
Van Goor, F.1
Yu, H.2
Burton, B.3
-
21
-
-
84943230777
-
Measurements of functional responses in human primary lung cells as a basis for personalized therapy for cystic fibrosis
-
Awatade NT, Uliyakina I, Farinha CM, et al. Measurements of functional responses in human primary lung cells as a basis for personalized therapy for cystic fibrosis. EBioMedicine 2015; 2: 147-153.
-
(2015)
EBioMedicine
, vol.2
, pp. 147-153
-
-
Awatade, N.T.1
Uliyakina, I.2
Farinha, C.M.3
-
22
-
-
84925778077
-
Rescue of NBD2 mutants N1303K and S1235R of CFTR by small-molecule correctors and transcomplementation
-
Rapino D, Sabirzhanova I, Lopes-Pacheco M, et al. Rescue of NBD2 mutants N1303K and S1235R of CFTR by small-molecule correctors and transcomplementation. PLoS One 2015; 10: e0119796.
-
(2015)
PLoS One
, vol.10
, pp. e0119796
-
-
Rapino, D.1
Sabirzhanova, I.2
Lopes-Pacheco, M.3
|