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Volumn 48, Issue 2, 2016, Pages 451-458

Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids

Author keywords

[No Author keywords available]

Indexed keywords

BITHIAZOLE DERIVATIVE; COMPOUND C1; COMPOUND C10; COMPOUND C11; COMPOUND C12; COMPOUND C13; COMPOUND C14; COMPOUND C15; COMPOUND C16; COMPOUND C17; COMPOUND C18; COMPOUND C2; COMPOUND C3; COMPOUND C4; COMPOUND C5; COMPOUND C6; COMPOUND C7; COMPOUND C8; COMPOUND C9; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; LUMACAFTOR; MUTANT PROTEIN; THIAZOLE DERIVATIVE; UNCLASSIFIED DRUG; 1,3 BENZODIOXOLE DERIVATIVE; AMINOPYRIDINE DERIVATIVE; CFTR PROTEIN, HUMAN;

EID: 84980016718     PISSN: 09031936     EISSN: 13993003     Source Type: Journal    
DOI: 10.1183/13993003.01192-2015     Document Type: Article
Times cited : (58)

References (22)
  • 1
    • 84890435909 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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 scopus 로고    scopus 로고
    • 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


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.