메뉴 건너뛰기




Volumn 283, Issue 2, 2016, Pages 246-264

Repairing the basic defect in cystic fibrosis - One approach is not enough

Author keywords

bypass approaches; correctors; cystic fibrosis; cystic fibrosis transmembrane conductance regulator; novel therapies; potentiators

Indexed keywords

ATALUREN; CHLORIDE; CHLORIDE CHANNEL; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; EPIGALLOCATECHIN GALLATE; EPITHELIAL SODIUM CHANNEL; EPITHELIAL SODIUM CHANNEL BLOCKING AGENT; FOSTAMATINIB; GENTAMICIN; IVACAFTOR; IVACAFTOR PLUS LUMACAFTOR; LIPOSOME; LUMACAFTOR; MERCAPTAMINE; N (3,5 DIAMINO 6 CHLOROPYRAZINE 2 CARBONYL) N' 4 [4 (2,3 DIHYDROXYPROPOXY)PHENYL]BUTYLGUANIDINE METHANESULFONATE; PLASMID DNA; PROTEIN KINASE SYK; PYRAZINE DERIVATIVE; QUINAZOLINE DERIVATIVE; TEZACAFTOR; UNCLASSIFIED DRUG; VRT 532; CFTR PROTEIN, HUMAN;

EID: 84969373509     PISSN: 1742464X     EISSN: 17424658     Source Type: Journal    
DOI: 10.1111/febs.13531     Document Type: Review
Times cited : (30)

References (136)
  • 1
    • 0036258208 scopus 로고    scopus 로고
    • Cystic fibrosis: A worldwide analysis of CFTR mutations-correlation with incidence data and application to screening
    • Bobadilla JL, Macek M Jr, Fine JP, &, Farrell PM, (2002) Cystic fibrosis: a worldwide analysis of CFTR mutations-correlation with incidence data and application to screening. Hum Mutat 19, 575-606.
    • (2002) Hum Mutat , vol.19 , pp. 575-606
    • Bobadilla, J.L.1    Macek, M.2    Fine, J.P.3    Farrell, P.M.4
  • 3
    • 15544371839 scopus 로고    scopus 로고
    • Assembly of functional CFTR chloride channels
    • Riordan JR, (2005) Assembly of functional CFTR chloride channels. Annu Rev Physiol 67, 701-718.
    • (2005) Annu Rev Physiol , vol.67 , pp. 701-718
    • Riordan, J.R.1
  • 4
    • 84918819788 scopus 로고    scopus 로고
    • New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls
    • Bell SC, De Boeck K, &, Amaral MD, (2015) New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls. Pharmacol Ther 145, 19-34.
    • (2015) Pharmacol Ther , vol.145 , pp. 19-34
    • Bell, S.C.1    De Boeck, K.2    Amaral, M.D.3
  • 5
    • 84883448606 scopus 로고    scopus 로고
    • Rescuing mutant CFTR: A multi-task approach to a better outcome in treating cystic fibrosis
    • Amaral MD, &, Farinha CM, (2013) Rescuing mutant CFTR: a multi-task approach to a better outcome in treating cystic fibrosis. Curr Pharm Des 19, 3497-3508.
    • (2013) Curr Pharm des , vol.19 , pp. 3497-3508
    • Amaral, M.D.1    Farinha, C.M.2
  • 6
    • 35548950947 scopus 로고    scopus 로고
    • The efficiency of nonsense-mediated mRNA decay is an inherent character and varies among different cells
    • Linde L, Boelz S, Neu-Yilik G, Kulozik AE, &, Kerem B, (2007a) The efficiency of nonsense-mediated mRNA decay is an inherent character and varies among different cells. Eur J Hum Genet 15, 1156-1162.
    • (2007) Eur J Hum Genet , vol.15 , pp. 1156-1162
    • Linde, L.1    Boelz, S.2    Neu-Yilik, G.3    Kulozik, A.E.4    Kerem, B.5
  • 8
    • 27744482654 scopus 로고    scopus 로고
    • The short apical membrane half-life of rescued {Delta}F508-cystic fibrosis transmembrane conductance regulator (CFTR) results from accelerated endocytosis of {Delta}F508-CFTR in polarized human airway epithelial cells
    • Swiatecka-Urban A, Brown A, Moreau-Marquis S, Renuka J, Coutermarsh B, Barnaby R, Karlson KH, Flotte TR, Fukuda M, Langford GM, et al,. (2005) The short apical membrane half-life of rescued {Delta}F508-cystic fibrosis transmembrane conductance regulator (CFTR) results from accelerated endocytosis of {Delta}F508-CFTR in polarized human airway epithelial cells. J Biol Chem 280, 36762-36772.
    • (2005) J Biol Chem , vol.280 , pp. 36762-36772
    • Swiatecka-Urban, A.1    Brown, A.2    Moreau-Marquis, S.3    Renuka, J.4    Coutermarsh, B.5    Barnaby, R.6    Karlson, K.H.7    Flotte, T.R.8    Fukuda, M.9    Langford, G.M.10
  • 9
    • 84874094442 scopus 로고    scopus 로고
    • HGF stimulation of Rac1 signaling enhances pharmacological correction of the most prevalent cystic fibrosis mutant F508del-CFTR
    • Moniz S, Sousa M, Moraes BJ, Mendes AI, Palma M, Barreto C, Fragata JI, Amaral MD, &, Matos P, (2013) HGF stimulation of Rac1 signaling enhances pharmacological correction of the most prevalent cystic fibrosis mutant F508del-CFTR. ACS Chem Biol 8, 432-442.
    • (2013) ACS Chem Biol , vol.8 , pp. 432-442
    • Moniz, S.1    Sousa, M.2    Moraes, B.J.3    Mendes, A.I.4    Palma, M.5    Barreto, C.6    Fragata, J.I.7    Amaral, M.D.8    Matos, P.9
  • 12
    • 0026656343 scopus 로고
    • Extensive posttranscriptional deletion of the coding sequences for part of nucleotide-binding fold 1 in respiratory epithelial mRNA transcripts of the cystic fibrosis transmembrane conductance regulator gene is not associated with the clinical manifestations of cystic fibrosis
    • Chu CS, Trapnell BC, Curristin SM, Cutting GR, &, Crystal RG, (1992) Extensive posttranscriptional deletion of the coding sequences for part of nucleotide-binding fold 1 in respiratory epithelial mRNA transcripts of the cystic fibrosis transmembrane conductance regulator gene is not associated with the clinical manifestations of cystic fibrosis. J Clin Invest 90, 785-790.
    • (1992) J Clin Invest , vol.90 , pp. 785-790
    • Chu, C.S.1    Trapnell, B.C.2    Curristin, S.M.3    Cutting, G.R.4    Crystal, R.G.5
  • 14
    • 60149095412 scopus 로고    scopus 로고
    • Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy
    • UK Cystic Fibrosis Gene Therapy Consortium
    • Griesenbach U, &, Alton EW, & UK Cystic Fibrosis Gene Therapy Consortium. (2009) Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy. Adv Drug Deliv Rev. 61, 128-139.
    • (2009) Adv Drug Deliv Rev , vol.61 , pp. 128-139
    • Griesenbach, U.1    Alton, E.W.2
  • 16
    • 0026781952 scopus 로고
    • Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive
    • Denning GM, Anderson MP, Amara JF, Marshall J, Smith AE, &, Welsh MJ, (1992) Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive. Nature 358, 761-764.
    • (1992) Nature , vol.358 , pp. 761-764
    • Denning, G.M.1    Anderson, M.P.2    Amara, J.F.3    Marshall, J.4    Smith, A.E.5    Welsh, M.J.6
  • 18
    • 33845197320 scopus 로고    scopus 로고
    • Revertant mutants G550E and 4RK rescue cystic fibrosis mutants in the first nucleotide-binding domain of CFTR by different mechanisms
    • Roxo-Rosa M, Xu Z, Schmidt A, Neto M, Cai Z, Soares CM, Sheppard DN, &, Amaral MD, (2006) Revertant mutants G550E and 4RK rescue cystic fibrosis mutants in the first nucleotide-binding domain of CFTR by different mechanisms. Proc Natl Acad Sci USA 103, 17891-17896.
    • (2006) Proc Natl Acad Sci USA , vol.103 , pp. 17891-17896
    • Roxo-Rosa, M.1    Xu, Z.2    Schmidt, A.3    Neto, M.4    Cai, Z.5    Soares, C.M.6    Sheppard, D.N.7    Amaral, M.D.8
  • 19
    • 84927176500 scopus 로고    scopus 로고
    • Cystic fibrosis genetics: From molecular understanding to clinical application
    • Cutting GR, (2015) Cystic fibrosis genetics: from molecular understanding to clinical application. Nat Rev Genet 16, 45-56.
    • (2015) Nat Rev Genet , vol.16 , pp. 45-56
    • Cutting, G.R.1
  • 20
    • 34547093657 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics: Old drugs and new therapeutic approaches
    • Hermann T, (2007) Aminoglycoside antibiotics: old drugs and new therapeutic approaches. Cell Mol Life Sci 64, 1841-1852.
    • (2007) Cell Mol Life Sci , vol.64 , pp. 1841-1852
    • Hermann, T.1
  • 23
    • 41149111377 scopus 로고    scopus 로고
    • PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model
    • Du M, Liu X, Welch EM, Hirawat S, Peltz SW, &, Bedwell DM, (2008) PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proc Natl Acad Sci USA 105, 2064-2069.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 2064-2069
    • Du, M.1    Liu, X.2    Welch, E.M.3    Hirawat, S.4    Peltz, S.W.5    Bedwell, D.M.6
  • 26
    • 84910627623 scopus 로고    scopus 로고
    • Ataluren: First global approval
    • Ryan NJ, (2014) Ataluren: first global approval. Drugs 74, 1709-1714.
    • (2014) Drugs , vol.74 , pp. 1709-1714
    • Ryan, N.J.1
  • 27
    • 0030042386 scopus 로고    scopus 로고
    • Glycerol reverses the misfolding phenotype of the most common cystic fibrosis mutation
    • Sato S, Ward CL, Krouse ME, Wine JJ, &, Kopito RR, (1996) Glycerol reverses the misfolding phenotype of the most common cystic fibrosis mutation. J Biol Chem 271, 635-638.
    • (1996) J Biol Chem , vol.271 , pp. 635-638
    • Sato, S.1    Ward, C.L.2    Krouse, M.E.3    Wine, J.J.4    Kopito, R.R.5
  • 28
    • 2942580461 scopus 로고    scopus 로고
    • CFTR and chaperones: Processing and degradation
    • Amaral MD, (2004) CFTR and chaperones: processing and degradation. J Mol Neurosci 23, 41-48.
    • (2004) J Mol Neurosci , vol.23 , pp. 41-48
    • Amaral, M.D.1
  • 29
    • 84863509037 scopus 로고    scopus 로고
    • Emergent properties of proteostasis in managing cystic fibrosis
    • Balch WE, Roth DM, &, Hutt DM, (2011) Emergent properties of proteostasis in managing cystic fibrosis. Cold Spring Harb Perspect Biol 3, a004499.
    • (2011) Cold Spring Harb Perspect Biol , vol.3 , pp. a004499
    • Balch, W.E.1    Roth, D.M.2    Hutt, D.M.3
  • 32
    • 84855202429 scopus 로고    scopus 로고
    • Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation
    • Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, et al,. (2011) Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax 67, 12-18.
    • (2011) Thorax , vol.67 , pp. 12-18
    • Clancy, J.P.1    Rowe, S.M.2    Accurso, F.J.3    Aitken, M.L.4    Amin, R.S.5    Ashlock, M.A.6    Ballmann, M.7    Boyle, M.P.8    Bronsveld, I.9    Campbell, P.W.10
  • 33
    • 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, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, &, Rodman D, (2014) 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 2, 527-538.
    • (2014) Lancet Respir Med , vol.2 , pp. 527-538
    • Boyle, M.P.1    Bell, S.C.2    Konstan, M.W.3    McColley, S.A.4    Rowe, S.M.5    Rietschel, E.6    Huang, X.7    Waltz, D.8    Patel, N.R.9    Rodman, D.10
  • 34
    • 33745282127 scopus 로고    scopus 로고
    • Specific rescue of cystic fibrosis transmembrane conductance regulator processing mutants using pharmacological chaperones
    • Wang Y, Bartlett MC, Loo TW, &, Clarke DM, (2006) Specific rescue of cystic fibrosis transmembrane conductance regulator processing mutants using pharmacological chaperones. Mol Pharmacol 70, 297-302.
    • (2006) Mol Pharmacol , vol.70 , pp. 297-302
    • Wang, Y.1    Bartlett, M.C.2    Loo, T.W.3    Clarke, D.M.4
  • 35
    • 33847122608 scopus 로고    scopus 로고
    • Modulating the folding of P-glycoprotein and cystic fibrosis transmembrane conductance regulator truncation mutants with pharmacological chaperones
    • Wang Y, Loo TW, Bartlett MC, &, Clarke DM, (2007a) Modulating the folding of P-glycoprotein and cystic fibrosis transmembrane conductance regulator truncation mutants with pharmacological chaperones. Mol Pharmacol 71, 751-758.
    • (2007) Mol Pharmacol , vol.71 , pp. 751-758
    • Wang, Y.1    Loo, T.W.2    Bartlett, M.C.3    Clarke, D.M.4
  • 36
    • 77950204636 scopus 로고    scopus 로고
    • Influence of cell background on pharmacological rescue of mutant CFTR
    • Pedemonte N, Tomati V, Sondo E, &, Galietta LJ, (2010) Influence of cell background on pharmacological rescue of mutant CFTR. Am J Physiol Cell Physiol 298, C866-C874.
    • (2010) Am J Physiol Cell Physiol , vol.298 , pp. C866-C874
    • Pedemonte, N.1    Tomati, V.2    Sondo, E.3    Galietta, L.J.4
  • 37
    • 84873436500 scopus 로고    scopus 로고
    • Correctors of {Delta}F508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein
    • He L, Kota P, Aleksandrov AA, Cui L, Jensen T, Dokholyan NV, &, Riordan JR, (2012) Correctors of {Delta}F508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein. FASEB J 27, 536-545.
    • (2012) FASEB J , vol.27 , pp. 536-545
    • He, L.1    Kota, P.2    Aleksandrov, A.A.3    Cui, L.4    Jensen, T.5    Dokholyan, N.V.6    Riordan, J.R.7
  • 39
    • 84877346547 scopus 로고    scopus 로고
    • Tgf-beta1 inhibits CFTR biogenesis and prevents functional rescue of DeltaF508-CFTR in primary differentiated human bronchial epithelial cells
    • Snodgrass SM, Cihil KM, Cornuet PK, Myerburg MM, &, Swiatecka-Urban A, (2013) Tgf-beta1 inhibits Cftr biogenesis and prevents functional rescue of DeltaF508-Cftr in primary differentiated human bronchial epithelial cells. PLoS One 8, e63167.
    • (2013) PLoS One , vol.8 , pp. e63167
    • Snodgrass, S.M.1    Cihil, K.M.2    Cornuet, P.K.3    Myerburg, M.M.4    Swiatecka-Urban, A.5
  • 41
    • 34548154971 scopus 로고    scopus 로고
    • Additive effect of multiple pharmacological chaperones on maturation of CFTR processing mutants
    • Wang Y, Loo TW, Bartlett MC, &, Clarke DM, (2007b) Additive effect of multiple pharmacological chaperones on maturation of CFTR processing mutants. Biochem J 406, 257-263.
    • (2007) Biochem J , vol.406 , pp. 257-263
    • Wang, Y.1    Loo, T.W.2    Bartlett, M.C.3    Clarke, D.M.4
  • 43
    • 84901453811 scopus 로고    scopus 로고
    • Synergy-based small-molecule screen using a human lung epithelial cell line yields DeltaF508-CFTR correctors that augment VX-809 maximal efficacy
    • Phuan PW, Veit G, Tan J, Roldan A, Finkbeiner WE, Lukacs GL, &, Verkman AS, (2014) Synergy-based small-molecule screen using a human lung epithelial cell line yields DeltaF508-CFTR correctors that augment VX-809 maximal efficacy. Mol Pharmacol 86, 42-51.
    • (2014) Mol Pharmacol , vol.86 , pp. 42-51
    • Phuan, P.W.1    Veit, G.2    Tan, J.3    Roldan, A.4    Finkbeiner, W.E.5    Lukacs, G.L.6    Verkman, A.S.7
  • 45
    • 85047689539 scopus 로고    scopus 로고
    • A genomic signature approach to rescue DeltaF508-cystic fibrosis transmembrane conductance regulator biosynthesis and function
    • Ramachandran S, Osterhaus SR, Karp PH, Welsh MJ, &, McCray PB Jr, (2014) A genomic signature approach to rescue DeltaF508-cystic fibrosis transmembrane conductance regulator biosynthesis and function. Am J Respir Cell Mol Biol 51, 354-362.
    • (2014) Am J Respir Cell Mol Biol , vol.51 , pp. 354-362
    • Ramachandran, S.1    Osterhaus, S.R.2    Karp, P.H.3    Welsh, M.J.4    McCray, P.B.5
  • 49
    • 24644464284 scopus 로고    scopus 로고
    • Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening
    • Pedemonte N, Lukacs GL, Du K, Caci E, Zegarra-Moran O, Galietta LJ, &, Verkman AS, (2005b) Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening. J Clin Invest 115, 2564-2571.
    • (2005) J Clin Invest , vol.115 , pp. 2564-2571
    • Pedemonte, N.1    Lukacs, G.L.2    Du, K.3    Caci, E.4    Zegarra-Moran, O.5    Galietta, L.J.6    Verkman, A.S.7
  • 52
    • 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, &, Hoffman BJ, (2014) Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros 13, 29-36.
    • (2014) J Cyst Fibros , vol.13 , pp. 29-36
    • Van Goor, F.1    Yu, H.2    Burton, B.3    Hoffman, B.J.4
  • 53
    • 84908227848 scopus 로고    scopus 로고
    • Structure-activity analysis of a CFTR channel potentiator: Distinct molecular parts underlie dual gating effects
    • Csanady L, &, Torocsik B, (2014) Structure-activity analysis of a CFTR channel potentiator: distinct molecular parts underlie dual gating effects. J Gen Physiol 144, 321-336.
    • (2014) J Gen Physiol , vol.144 , pp. 321-336
    • Csanady, L.1    Torocsik, B.2
  • 59
    • 84969374206 scopus 로고    scopus 로고
    • Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR
    • Farinha CM, Sousa M, Canato S, Schmidt A, Uliyakina I, &, Amaral MD, (2015) Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR. Pharmacol Res Perspect 3, e00152.
    • (2015) Pharmacol Res Perspect , vol.3 , pp. e00152
    • Farinha, C.M.1    Sousa, M.2    Canato, S.3    Schmidt, A.4    Uliyakina, I.5    Amaral, M.D.6
  • 60
    • 55549094466 scopus 로고    scopus 로고
    • Multiple membrane-cytoplasmic domain contacts in the cystic fibrosis transmembrane conductance regulator (CFTR) mediate regulation of channel gating
    • He L, Aleksandrov AA, Serohijos AW, Hegedus T, Aleksandrov LA, Cui L, Dokholyan NV, &, Riordan JR, (2008) Multiple membrane-cytoplasmic domain contacts in the cystic fibrosis transmembrane conductance regulator (CFTR) mediate regulation of channel gating. J Biol Chem 283, 26383-26390.
    • (2008) J Biol Chem , vol.283 , pp. 26383-26390
    • He, L.1    Aleksandrov, A.A.2    Serohijos, A.W.3    Hegedus, T.4    Aleksandrov, L.A.5    Cui, L.6    Dokholyan, N.V.7    Riordan, J.R.8
  • 61
    • 77957302946 scopus 로고    scopus 로고
    • Thermal unfolding studies show the disease causing F508del mutation in CFTR thermodynamically destabilizes nucleotide-binding domain 1
    • Protasevich I, Yang Z, Wang C, Atwell S, Zhao X, Emtage S, Wetmore D, Hunt JF, &, Brouillette CG, (2010) Thermal unfolding studies show the disease causing F508del mutation in CFTR thermodynamically destabilizes nucleotide-binding domain 1. Protein Sci 19, 1917-1931.
    • (2010) Protein Sci , vol.19 , pp. 1917-1931
    • Protasevich, I.1    Yang, Z.2    Wang, C.3    Atwell, S.4    Zhao, X.5    Emtage, S.6    Wetmore, D.7    Hunt, J.F.8    Brouillette, C.G.9
  • 63
    • 84933046182 scopus 로고    scopus 로고
    • Decoding F508del misfolding in cystic fibrosis
    • Wang XR, &, Li C, (2014) Decoding F508del misfolding in cystic fibrosis. Biomolecules 4, 498-509.
    • (2014) Biomolecules , vol.4 , pp. 498-509
    • Wang, X.R.1    Li, C.2
  • 66
    • 78649775607 scopus 로고    scopus 로고
    • The primary folding defect and rescue of DeltaF508 CFTR emerge during translation of the mutant domain
    • Hoelen H, Kleizen B, Schmidt A, Richardson J, Charitou P, Thomas PJ, &, Braakman I, (2010) The primary folding defect and rescue of DeltaF508 CFTR emerge during translation of the mutant domain. PLoS One 5, e15458.
    • (2010) PLoS One , vol.5 , pp. e15458
    • Hoelen, H.1    Kleizen, B.2    Schmidt, A.3    Richardson, J.4    Charitou, P.5    Thomas, P.J.6    Braakman, I.7
  • 67
    • 84883465161 scopus 로고    scopus 로고
    • Control of CFTR membrane trafficking: Not just from the ER to the Golgi
    • Farinha CM, Matos P, &, Amaral MD, (2013b) Control of CFTR membrane trafficking: not just from the ER to the Golgi. FEBS J 280, 4396-4406.
    • (2013) FEBS J , vol.280 , pp. 4396-4406
    • Farinha, C.M.1    Matos, P.2    Amaral, M.D.3
  • 69
    • 64049096002 scopus 로고    scopus 로고
    • N-glycans are direct determinants of CFTR folding and stability in secretory and endocytic membrane traffic
    • Glozman R, Okiyoneda T, Mulvihill CM, Rini JM, Barriere H, &, Lukacs GL, (2009) N-glycans are direct determinants of CFTR folding and stability in secretory and endocytic membrane traffic. J Cell Biol 184, 847-862.
    • (2009) J Cell Biol , vol.184 , pp. 847-862
    • Glozman, R.1    Okiyoneda, T.2    Mulvihill, C.M.3    Rini, J.M.4    Barriere, H.5    Lukacs, G.L.6
  • 70
    • 0030712030 scopus 로고    scopus 로고
    • Constitutive internalization of cystic fibrosis transmembrane conductance regulator occurs via clathrin-dependent endocytosis and is regulated by protein phosphorylation
    • Lukacs GL, Segal G, Kartner N, Grinstein S, &, Zhang F, (1997) Constitutive internalization of cystic fibrosis transmembrane conductance regulator occurs via clathrin-dependent endocytosis and is regulated by protein phosphorylation. Biochem J 328, 353-361.
    • (1997) Biochem J , vol.328 , pp. 353-361
    • Lukacs, G.L.1    Segal, G.2    Kartner, N.3    Grinstein, S.4    Zhang, F.5
  • 71
    • 78049497975 scopus 로고    scopus 로고
    • Quality control for unfolded proteins at the plasma membrane
    • Apaja PM, Xu H, &, Lukacs GL, (2010) Quality control for unfolded proteins at the plasma membrane. J Cell Biol 191, 553-570.
    • (2010) J Cell Biol , vol.191 , pp. 553-570
    • Apaja, P.M.1    Xu, H.2    Lukacs, G.L.3
  • 72
    • 84879866418 scopus 로고    scopus 로고
    • Regulated recycling of mutant CFTR is partially restored by pharmacological treatment
    • Holleran JP, Zeng J, Frizzell RA, &, Watkins SC, (2013) Regulated recycling of mutant CFTR is partially restored by pharmacological treatment. J Cell Sci 126, 2692-2703.
    • (2013) J Cell Sci , vol.126 , pp. 2692-2703
    • Holleran, J.P.1    Zeng, J.2    Frizzell, R.A.3    Watkins, S.C.4
  • 74
    • 77249126439 scopus 로고    scopus 로고
    • Modulation of endocytic trafficking and apical stability of CFTR in primary human airway epithelial cultures
    • Cholon DM, O'Neal WK, Randell SH, Riordan JR, &, Gentzsch M, (2010) Modulation of endocytic trafficking and apical stability of CFTR in primary human airway epithelial cultures. Am J Physiol Lung Cell Mol Physiol 298, L304-L314.
    • (2010) Am J Physiol Lung Cell Mol Physiol , vol.298 , pp. L304-L314
    • Cholon, D.M.1    O'Neal, W.K.2    Randell, S.H.3    Riordan, J.R.4    Gentzsch, M.5
  • 77
    • 13544273548 scopus 로고    scopus 로고
    • Regulation of cystic fibrosis transmembrane regulator trafficking and protein expression by a Rho family small GTPase TC10
    • Cheng J, Wang H, &, Guggino WB, (2005) Regulation of cystic fibrosis transmembrane regulator trafficking and protein expression by a Rho family small GTPase TC10. J Biol Chem 280, 3731-3739.
    • (2005) J Biol Chem , vol.280 , pp. 3731-3739
    • Cheng, J.1    Wang, H.2    Guggino, W.B.3
  • 78
    • 53349151733 scopus 로고    scopus 로고
    • Role of N-linked oligosaccharides in the biosynthetic processing of the cystic fibrosis membrane conductance regulator
    • Chang XB, Mengos A, Hou YX, Cui L, Jensen TJ, Aleksandrov A, Riordan JR, &, Gentzsch M, (2008) Role of N-linked oligosaccharides in the biosynthetic processing of the cystic fibrosis membrane conductance regulator. J Cell Sci 121, 2814-2823.
    • (2008) J Cell Sci , vol.121 , pp. 2814-2823
    • Chang, X.B.1    Mengos, A.2    Hou, Y.X.3    Cui, L.4    Jensen, T.J.5    Aleksandrov, A.6    Riordan, J.R.7    Gentzsch, M.8
  • 79
    • 0347717877 scopus 로고    scopus 로고
    • Modulation of mature cystic fibrosis transmembrane regulator protein by the PDZ domain protein CAL
    • Cheng J, Wang H, &, Guggino WB, (2004) Modulation of mature cystic fibrosis transmembrane regulator protein by the PDZ domain protein CAL. J Biol Chem 279, 1892-1898.
    • (2004) J Biol Chem , vol.279 , pp. 1892-1898
    • Cheng, J.1    Wang, H.2    Guggino, W.B.3
  • 80
    • 52249089383 scopus 로고    scopus 로고
    • The relative binding affinities of PDZ partners for CFTR: A biochemical basis for efficient endocytic recycling
    • Cushing PR, Fellows A, Villone D, Boisguerin P, &, Madden DR, (2008) The relative binding affinities of PDZ partners for CFTR: a biochemical basis for efficient endocytic recycling. Biochemistry 47, 10084-10098.
    • (2008) Biochemistry , vol.47 , pp. 10084-10098
    • Cushing, P.R.1    Fellows, A.2    Villone, D.3    Boisguerin, P.4    Madden, D.R.5
  • 81
    • 75649086188 scopus 로고    scopus 로고
    • Na+/H+ exchanger regulatory factor 1 overexpression-dependent increase of cytoskeleton organization is fundamental in the rescue of F508del cystic fibrosis transmembrane conductance regulator in human airway CFBE41o- cells
    • Favia M, Guerra L, Fanelli T, Cardone RA, Monterisi S, Di Sole F, Castellani S, Chen M, Seidler U, Reshkin SJ, et al,. (2010) Na+/H+ exchanger regulatory factor 1 overexpression-dependent increase of cytoskeleton organization is fundamental in the rescue of F508del cystic fibrosis transmembrane conductance regulator in human airway CFBE41o- cells. Mol Biol Cell 21, 73-86.
    • (2010) Mol Biol Cell , vol.21 , pp. 73-86
    • Favia, M.1    Guerra, L.2    Fanelli, T.3    Cardone, R.A.4    Monterisi, S.5    Di Sole, F.6    Castellani, S.7    Chen, M.8    Seidler, U.9    Reshkin, S.J.10
  • 82
    • 28844474806 scopus 로고    scopus 로고
    • Na+/H+ exchanger regulatory factor isoform 1 overexpression modulates cystic fibrosis transmembrane conductance regulator (CFTR) expression and activity in human airway 16HBE14o- cells and rescues DeltaF508 CFTR functional expression in cystic fibrosis cells
    • Guerra L, Fanelli T, Favia M, Riccardi SM, Busco G, Cardone RA, Carrabino S, Weinman EJ, Reshkin SJ, Conese M, et al,. (2005) Na+/H+ exchanger regulatory factor isoform 1 overexpression modulates cystic fibrosis transmembrane conductance regulator (CFTR) expression and activity in human airway 16HBE14o- cells and rescues DeltaF508 CFTR functional expression in cystic fibrosis cells. J Biol Chem 280, 40925-40933.
    • (2005) J Biol Chem , vol.280 , pp. 40925-40933
    • Guerra, L.1    Fanelli, T.2    Favia, M.3    Riccardi, S.M.4    Busco, G.5    Cardone, R.A.6    Carrabino, S.7    Weinman, E.J.8    Reshkin, S.J.9    Conese, M.10
  • 83
    • 84868549245 scopus 로고    scopus 로고
    • NHERF1 and CFTR restore tight junction organisation and function in cystic fibrosis airway epithelial cells: Role of ezrin and the RhoA/ROCK pathway
    • Castellani S, Guerra L, Favia M, Di Gioia S, Casavola V, &, Conese M, (2012) NHERF1 and CFTR restore tight junction organisation and function in cystic fibrosis airway epithelial cells: role of ezrin and the RhoA/ROCK pathway. Lab Invest 92, 1527-1540.
    • (2012) Lab Invest , vol.92 , pp. 1527-1540
    • Castellani, S.1    Guerra, L.2    Favia, M.3    Di Gioia, S.4    Casavola, V.5    Conese, M.6
  • 84
    • 84929667112 scopus 로고    scopus 로고
    • A NHERF1 PDZ domain switch prevents CHIP Ub ligase-mediated peripheral protein quality control retaining misfolded CFTR at the cell surface
    • Loureiro CA, Matos AM, Dias-Alves Â, Pereira JF, Uliyakina I, Barros P, Amaral MD, &, Matos P, (2015) A NHERF1 PDZ domain switch prevents CHIP Ub ligase-mediated peripheral protein quality control retaining misfolded CFTR at the cell surface. Sci Signal 8, ra48.
    • (2015) Sci Signal , vol.8 , pp. ra48
    • Loureiro, C.A.1    Matos, A.M.2    Dias-Alves, Â.3    Pereira, J.F.4    Uliyakina, I.5    Barros, P.6    Amaral, M.D.7    Matos, P.8
  • 85
    • 33845460799 scopus 로고    scopus 로고
    • Tracking of quantum dot-labeled CFTR shows near immobilization by C-terminal PDZ interactions
    • Haggie PM, Kim JK, Lukacs GL, &, Verkman AS, (2006) Tracking of quantum dot-labeled CFTR shows near immobilization by C-terminal PDZ interactions. Mol Biol Cell 17, 4937-4945.
    • (2006) Mol Biol Cell , vol.17 , pp. 4937-4945
    • Haggie, P.M.1    Kim, J.K.2    Lukacs, G.L.3    Verkman, A.S.4
  • 87
    • 27844570909 scopus 로고    scopus 로고
    • Ezrin controls the macromolecular complexes formed between an adapter protein Na+/H+ exchanger regulatory factor and the cystic fibrosis transmembrane conductance regulator
    • Li J, Dai Z, Jana D, Callaway DJ, &, Bu Z, (2005) Ezrin controls the macromolecular complexes formed between an adapter protein Na+/H+ exchanger regulatory factor and the cystic fibrosis transmembrane conductance regulator. J Biol Chem 280, 37634-37643.
    • (2005) J Biol Chem , vol.280 , pp. 37634-37643
    • Li, J.1    Dai, Z.2    Jana, D.3    Callaway, D.J.4    Bu, Z.5
  • 88
    • 33846328758 scopus 로고    scopus 로고
    • CFTR surface expression and chloride currents are decreased by inhibitors of N-WASP and actin polymerization
    • Ganeshan R, Nowotarski K, Di A, Nelson DJ, &, Kirk KL, (2007) CFTR surface expression and chloride currents are decreased by inhibitors of N-WASP and actin polymerization. Biochim Biophys Acta 1773, 192-200.
    • (2007) Biochim Biophys Acta , vol.1773 , pp. 192-200
    • Ganeshan, R.1    Nowotarski, K.2    Di, A.3    Nelson, D.J.4    Kirk, K.L.5
  • 89
    • 84876269644 scopus 로고    scopus 로고
    • Local modulation of cystic fibrosis conductance regulator: Cytoskeleton and compartmentalized cAMP signalling
    • Monterisi S, Casavola V, &, Zaccolo M, (2013) Local modulation of cystic fibrosis conductance regulator: cytoskeleton and compartmentalized cAMP signalling. Br J Pharmacol 169, 1-9.
    • (2013) Br J Pharmacol , vol.169 , pp. 1-9
    • Monterisi, S.1    Casavola, V.2    Zaccolo, M.3
  • 90
    • 84861362217 scopus 로고    scopus 로고
    • CFTR regulation in human airway epithelial cells requires integrity of the actin cytoskeleton and compartmentalized cAMP and PKA activity
    • Monterisi S, Favia M, Guerra L, Cardone RA, Marzulli D, Reshkin SJ, Casavola V, &, Zaccolo M, (2012) CFTR regulation in human airway epithelial cells requires integrity of the actin cytoskeleton and compartmentalized cAMP and PKA activity. J Cell Sci 125, 1106-1117.
    • (2012) J Cell Sci , vol.125 , pp. 1106-1117
    • Monterisi, S.1    Favia, M.2    Guerra, L.3    Cardone, R.A.4    Marzulli, D.5    Reshkin, S.J.6    Casavola, V.7    Zaccolo, M.8
  • 91
    • 78650770246 scopus 로고    scopus 로고
    • Hepatocyte growth factor twenty years on: Much more than a growth factor
    • Nakamura T, Sakai K, Nakamura T, &, Matsumoto K, (2011) Hepatocyte growth factor twenty years on: much more than a growth factor. J Gastroenterol Hepatol 26 (Suppl 1), 188-202.
    • (2011) J Gastroenterol Hepatol , vol.26 , pp. 188-202
    • Nakamura, T.1    Sakai, K.2    Nakamura, T.3    Matsumoto, K.4
  • 92
    • 78651099581 scopus 로고    scopus 로고
    • Hepatocyte growth factor in lung repair and pulmonary fibrosis
    • Panganiban RA, &, Day RM, (2011) Hepatocyte growth factor in lung repair and pulmonary fibrosis. Acta Pharmacol Sin 32, 12-20.
    • (2011) Acta Pharmacol Sin , vol.32 , pp. 12-20
    • Panganiban, R.A.1    Day, R.M.2
  • 93
    • 84875125485 scopus 로고    scopus 로고
    • Hepatocyte growth factor is an attractive target for the treatment of pulmonary fibrosis
    • Chakraborty S, Chopra P, Hak A, Dastidar SG, &, Ray A, (2013) Hepatocyte growth factor is an attractive target for the treatment of pulmonary fibrosis. Expert Opin Investig Drugs 22, 499-515.
    • (2013) Expert Opin Investig Drugs , vol.22 , pp. 499-515
    • Chakraborty, S.1    Chopra, P.2    Hak, A.3    Dastidar, S.G.4    Ray, A.5
  • 94
    • 84902466769 scopus 로고    scopus 로고
    • Emerging molecular targets in oncology: Clinical potential of MET/hepatocyte growth-factor inhibitors
    • Smyth EC, Sclafani F, &, Cunningham D, (2014) Emerging molecular targets in oncology: clinical potential of MET/hepatocyte growth-factor inhibitors. Onco Targets Ther 7, 1001-1014.
    • (2014) Onco Targets Ther , vol.7 , pp. 1001-1014
    • Smyth, E.C.1    Sclafani, F.2    Cunningham, D.3
  • 95
    • 33745772850 scopus 로고    scopus 로고
    • New insights into cystic fibrosis: Molecular switches that regulate CFTR
    • Guggino WB, &, Stanton BA, (2006) New insights into cystic fibrosis: molecular switches that regulate CFTR. Nat Rev Mol Cell Biol 7, 426-436.
    • (2006) Nat Rev Mol Cell Biol , vol.7 , pp. 426-436
    • Guggino, W.B.1    Stanton, B.A.2
  • 96
    • 84883445123 scopus 로고    scopus 로고
    • CFTR: A hub for kinases and cross-talk of cAMP and Ca
    • Kunzelmann K, &, Mehta A, (2013) CFTR: a hub for kinases and cross-talk of cAMP and Ca. FEBS J 280, 4417-4429.
    • (2013) FEBS J , vol.280 , pp. 4417-4429
    • Kunzelmann, K.1    Mehta, A.2
  • 98
    • 80053611644 scopus 로고    scopus 로고
    • Antagonistic regulation of CFTR cell surface expression by protein kinases WNK4 and spleen tyrosine kinase
    • Mendes AI, Matos P, Moniz S, Luz S, Amaral MD, Farinha CM, &, Jordan P, (2011) Antagonistic regulation of CFTR cell surface expression by protein kinases WNK4 and spleen tyrosine kinase. Mol Cell Biol 31, 4076-4086.
    • (2011) Mol Cell Biol , vol.31 , pp. 4076-4086
    • Mendes, A.I.1    Matos, P.2    Moniz, S.3    Luz, S.4    Amaral, M.D.5    Farinha, C.M.6    Jordan, P.7
  • 99
    • 84905459655 scopus 로고    scopus 로고
    • Getting Syk: Spleen tyrosine kinase as a therapeutic target
    • Geahlen RL, (2014) Getting Syk: spleen tyrosine kinase as a therapeutic target. Trends Pharmacol Sci 35, 414-422.
    • (2014) Trends Pharmacol Sci , vol.35 , pp. 414-422
    • Geahlen, R.L.1
  • 101
    • 84856023801 scopus 로고    scopus 로고
    • Specific inhibition of spleen tyrosine kinase suppresses leukocyte immune function and inflammation in animal models of rheumatoid arthritis
    • Coffey G, DeGuzman F, Inagaki M, Pak Y, Delaney SM, Ives D, Betz A, Jia ZJ, Pandey A, Baker D, et al,. (2012) Specific inhibition of spleen tyrosine kinase suppresses leukocyte immune function and inflammation in animal models of rheumatoid arthritis. J Pharmacol Exp Ther 340, 350-359.
    • (2012) J Pharmacol Exp Ther , vol.340 , pp. 350-359
    • Coffey, G.1    DeGuzman, F.2    Inagaki, M.3    Pak, Y.4    Delaney, S.M.5    Ives, D.6    Betz, A.7    Jia, Z.J.8    Pandey, A.9    Baker, D.10
  • 102
    • 84886741044 scopus 로고    scopus 로고
    • Selective inhibition of spleen tyrosine kinase (SYK) with a novel orally bioavailable small molecule inhibitor, RO9021, impinges on various innate and adaptive immune responses: Implications for SYK inhibitors in autoimmune disease therapy
    • Liao C, Hsu J, Kim Y, Hu DQ, Xu D, Zhang J, Pashine A, Menke J, Whittard T, Romero N, et al,. (2013) Selective inhibition of spleen tyrosine kinase (SYK) with a novel orally bioavailable small molecule inhibitor, RO9021, impinges on various innate and adaptive immune responses: implications for SYK inhibitors in autoimmune disease therapy. Arthritis Res Ther 15, R146.
    • (2013) Arthritis Res Ther , vol.15 , pp. R146
    • Liao, C.1    Hsu, J.2    Kim, Y.3    Hu, D.Q.4    Xu, D.5    Zhang, J.6    Pashine, A.7    Menke, J.8    Whittard, T.9    Romero, N.10
  • 104
    • 84875845688 scopus 로고    scopus 로고
    • Effects of fostamatinib (R788), an oral spleen tyrosine kinase inhibitor, on health-related quality of life in patients with active rheumatoid arthritis: Analyses of patient-reported outcomes from a randomized, double-blind, placebo-controlled trial
    • Weinblatt ME, Kavanaugh A, Genovese MC, Jones DA, Musser TK, Grossbard EB, &, Magilavy DB, (2013) Effects of fostamatinib (R788), an oral spleen tyrosine kinase inhibitor, on health-related quality of life in patients with active rheumatoid arthritis: analyses of patient-reported outcomes from a randomized, double-blind, placebo-controlled trial. J Rheumatol 40, 369-378.
    • (2013) J Rheumatol , vol.40 , pp. 369-378
    • Weinblatt, M.E.1    Kavanaugh, A.2    Genovese, M.C.3    Jones, D.A.4    Musser, T.K.5    Grossbard, E.B.6    Magilavy, D.B.7
  • 106
    • 77955921530 scopus 로고    scopus 로고
    • Regulation and function of potassium channels in aldosterone-sensitive distal nephron
    • Wang WH, Yue P, Sun P, &, Lin DH, (2010b) Regulation and function of potassium channels in aldosterone-sensitive distal nephron. Curr Opin Nephrol Hypertens 19, 463-470.
    • (2010) Curr Opin Nephrol Hypertens , vol.19 , pp. 463-470
    • Wang, W.H.1    Yue, P.2    Sun, P.3    Lin, D.H.4
  • 107
    • 84919808164 scopus 로고    scopus 로고
    • Activation of 3-phosphoinositide-dependent kinase 1 (PDK1) and serum- and glucocorticoid-induced protein kinase 1 (SGK1) by short-chain sphingolipid C4-ceramide rescues the trafficking defect of DeltaF508-cystic fibrosis transmembrane conductance regulator (DeltaF508-CFTR)
    • Caohuy H, Yang Q, Eudy Y, Ha TA, Xu AE, Glover M, Frizzell RA, Jozwik C, &, Pollard HB, (2014) Activation of 3-phosphoinositide-dependent kinase 1 (PDK1) and serum- and glucocorticoid-induced protein kinase 1 (SGK1) by short-chain sphingolipid C4-ceramide rescues the trafficking defect of DeltaF508-cystic fibrosis transmembrane conductance regulator (DeltaF508-CFTR). J Biol Chem 289, 35953-35968.
    • (2014) J Biol Chem , vol.289 , pp. 35953-35968
    • Caohuy, H.1    Yang, Q.2    Eudy, Y.3    Ha, T.A.4    Xu, A.E.5    Glover, M.6    Frizzell, R.A.7    Jozwik, C.8    Pollard, H.B.9
  • 111
    • 77952925551 scopus 로고    scopus 로고
    • Ceramide in Pseudomonas aeruginosa infections and cystic fibrosis
    • Becker KA, Grassme H, Zhang Y, &, Gulbins E, (2010) Ceramide in Pseudomonas aeruginosa infections and cystic fibrosis. Cell Physiol Biochem 26, 57-66.
    • (2010) Cell Physiol Biochem , vol.26 , pp. 57-66
    • Becker, K.A.1    Grassme, H.2    Zhang, Y.3    Gulbins, E.4
  • 112
  • 113
    • 0033104582 scopus 로고    scopus 로고
    • CFTR, a regulator of channels
    • Kunzelmann K, &, Schreiber R, (1999) CFTR, a regulator of channels. J Membr Biol 168, 1-8.
    • (1999) J Membr Biol , vol.168 , pp. 1-8
    • Kunzelmann, K.1    Schreiber, R.2
  • 114
    • 0034919237 scopus 로고    scopus 로고
    • Mechanisms of the inhibition of epithelial Na(+) channels by CFTR and purinergic stimulation
    • Kunzelmann K, Schreiber R, &, Boucherot A, (2001) Mechanisms of the inhibition of epithelial Na(+) channels by CFTR and purinergic stimulation. Kidney Int 60, 455-461.
    • (2001) Kidney Int , vol.60 , pp. 455-461
    • Kunzelmann, K.1    Schreiber, R.2    Boucherot, A.3
  • 115
    • 34247330452 scopus 로고    scopus 로고
    • Regulatory interactions of N1303K-CFTR and ENaC in Xenopus oocytes: Evidence that chloride transport is not necessary for inhibition of ENaC
    • Suaud L, Yan W, Carattino MD, Robay A, Kleyman TR, &, Rubenstein RC, (2007) Regulatory interactions of N1303K-CFTR and ENaC in Xenopus oocytes: evidence that chloride transport is not necessary for inhibition of ENaC. Am J Physiol Cell Physiol 292, C1553-C1561.
    • (2007) Am J Physiol Cell Physiol , vol.292 , pp. C1553-C1561
    • Suaud, L.1    Yan, W.2    Carattino, M.D.3    Robay, A.4    Kleyman, T.R.5    Rubenstein, R.C.6
  • 117
    • 0035163461 scopus 로고    scopus 로고
    • Pharmacotherapy of the ion transport defect in cystic fibrosis
    • Kunzelmann K, &, Mall M, (2001) Pharmacotherapy of the ion transport defect in cystic fibrosis. Clin Exp Pharmacol Physiol 28, 857-867.
    • (2001) Clin Exp Pharmacol Physiol , vol.28 , pp. 857-867
    • Kunzelmann, K.1    Mall, M.2
  • 118
    • 0025274624 scopus 로고
    • Acute and long-term amiloride inhalation in cystic fibrosis lung disease. A rational approach to cystic fibrosis therapy
    • App EM, King M, Helfesrieder R, Kohler D, &, Matthys H, (1990) Acute and long-term amiloride inhalation in cystic fibrosis lung disease. A rational approach to cystic fibrosis therapy. Am Rev Respir Dis 141, 605-612.
    • (1990) Am Rev Respir Dis , vol.141 , pp. 605-612
    • App, E.M.1    King, M.2    Helfesrieder, R.3    Kohler, D.4    Matthys, H.5
  • 119
    • 41149164824 scopus 로고    scopus 로고
    • Pharmacological properties of N-(3,5-diamino-6-chloropyrazine-2-carbonyl)-N'-4-[4-(2,3-dihydroxypropoxy)phenyl] butyl-guanidine methanesulfonate (552-02), a novel epithelial sodium channel blocker with potential clinical efficacy for cystic fibrosis lung disease
    • Hirsh AJ, Zhang J, Zamurs A, Fleegle J, Thelin WR, Caldwell RA, Sabater JR, Abraham WM, Donowitz M, Cha B, et al,. (2008) Pharmacological properties of N-(3,5-diamino-6-chloropyrazine-2-carbonyl)-N'-4-[4-(2,3-dihydroxypropoxy)phenyl] butyl-guanidine methanesulfonate (552-02), a novel epithelial sodium channel blocker with potential clinical efficacy for cystic fibrosis lung disease. J Pharmacol Exp Ther 325, 77-88.
    • (2008) J Pharmacol Exp Ther , vol.325 , pp. 77-88
    • Hirsh, A.J.1    Zhang, J.2    Zamurs, A.3    Fleegle, J.4    Thelin, W.R.5    Caldwell, R.A.6    Sabater, J.R.7    Abraham, W.M.8    Donowitz, M.9    Cha, B.10
  • 120
    • 85050578543 scopus 로고    scopus 로고
    • ENaC inhibitors for the treatment of cystic fibrosis
    • Butler R, Hunt T, &, Smith NJ, (2015) ENaC inhibitors for the treatment of cystic fibrosis. Pharm Pat Anal 4, 17-27.
    • (2015) Pharm Pat Anal , vol.4 , pp. 17-27
    • Butler, R.1    Hunt, T.2    Smith, N.J.3
  • 123
    • 0025874599 scopus 로고
    • Activation by extracellular nucleotides of chloride secretion in the airway epithelia of patients with cystic fibrosis
    • Knowles MR, Clarke LL, &, Boucher RC, (1991) Activation by extracellular nucleotides of chloride secretion in the airway epithelia of patients with cystic fibrosis. N Engl J Med 325, 533-538.
    • (1991) N Engl J Med , vol.325 , pp. 533-538
    • Knowles, M.R.1    Clarke, L.L.2    Boucher, R.C.3
  • 125
    • 51549120559 scopus 로고    scopus 로고
    • Expression cloning of TMEM16A as a calcium-activated chloride channel subunit
    • Schroeder BC, Cheng T, Jan YN, &, Jan LY, (2008) Expression cloning of TMEM16A as a calcium-activated chloride channel subunit. Cell 134, 1019-1029.
    • (2008) Cell , vol.134 , pp. 1019-1029
    • Schroeder, B.C.1    Cheng, T.2    Jan, Y.N.3    Jan, L.Y.4
  • 129
    • 80355129636 scopus 로고    scopus 로고
    • Small-molecule activators of TMEM16A, a calcium-activated chloride channel, stimulate epithelial chloride secretion and intestinal contraction
    • Namkung W, Yao Z, Finkbeiner WE, &, Verkman AS, (2011) Small-molecule activators of TMEM16A, a calcium-activated chloride channel, stimulate epithelial chloride secretion and intestinal contraction. FASEB J 25, 4048-4062.
    • (2011) FASEB J , vol.25 , pp. 4048-4062
    • Namkung, W.1    Yao, Z.2    Finkbeiner, W.E.3    Verkman, A.S.4
  • 130
    • 84898046222 scopus 로고    scopus 로고
    • Development and validation of HTS assay for screening the calcium-activated chloride channel modulators in TMEM16A stably expressed CHO cells
    • Qi J, Wang Y, Liu Y, Zhang F, Guan B, &, Zhang H, (2014) Development and validation of HTS assay for screening the calcium-activated chloride channel modulators in TMEM16A stably expressed CHO cells. Anal Bioanal Chem 406, 1713-1721.
    • (2014) Anal Bioanal Chem , vol.406 , pp. 1713-1721
    • Qi, J.1    Wang, Y.2    Liu, Y.3    Zhang, F.4    Guan, B.5    Zhang, H.6
  • 131
    • 84857478183 scopus 로고    scopus 로고
    • Differential contribution of SLC26A9 to Cl(-) conductance in polarized and non-polarized epithelial cells
    • Ousingsawat J, Schreiber R, &, Kunzelmann K, (2012) Differential contribution of SLC26A9 to Cl(-) conductance in polarized and non-polarized epithelial cells. J Cell Physiol 227, 2323-2329.
    • (2012) J Cell Physiol , vol.227 , pp. 2323-2329
    • Ousingsawat, J.1    Schreiber, R.2    Kunzelmann, K.3
  • 132
    • 80052328287 scopus 로고    scopus 로고
    • The K+ channel opener 1-EBIO potentiates residual function of mutant CFTR in rectal biopsies from cystic fibrosis patients
    • Roth EK, Hirtz S, Duerr J, Wenning D, Eichler I, Seydewitz HH, Amaral MD, &, Mall MA, (2011) The K+ channel opener 1-EBIO potentiates residual function of mutant CFTR in rectal biopsies from cystic fibrosis patients. PLoS One 6, e24445.
    • (2011) PLoS One , vol.6 , pp. e24445
    • Roth, E.K.1    Hirtz, S.2    Duerr, J.3    Wenning, D.4    Eichler, I.5    Seydewitz, H.H.6    Amaral, M.D.7    Mall, M.A.8
  • 133
    • 84869389296 scopus 로고    scopus 로고
    • Targeting autophagy as a novel strategy for facilitating the therapeutic action of potentiators on DeltaF508 cystic fibrosis transmembrane conductance regulator
    • Luciani A, Villella VR, Esposito S, Gavina M, Russo I, Silano M, Guido S, Pettoello-Mantovani M, Carnuccio R, Scholte B, et al,. (2012) Targeting autophagy as a novel strategy for facilitating the therapeutic action of potentiators on DeltaF508 cystic fibrosis transmembrane conductance regulator. Autophagy 8, 1657-1672.
    • (2012) Autophagy , vol.8 , pp. 1657-1672
    • Luciani, A.1    Villella, V.R.2    Esposito, S.3    Gavina, M.4    Russo, I.5    Silano, M.6    Guido, S.7    Pettoello-Mantovani, M.8    Carnuccio, R.9    Scholte, B.10
  • 136
    • 84969380597 scopus 로고    scopus 로고
    • Cystic fibrosis drug ivacaftor offers patients new hope
    • 2 November 2011
    • Khan A, (2011) Cystic fibrosis drug ivacaftor offers patients new hope, Los Angeles Times, 2 November 2011. http://www.latimes.com/health/la-he-cystic-fibrosis-drug-20111103-story.html.
    • (2011) Los Angeles Times
    • Khan, A.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.