메뉴 건너뛰기




Volumn 16, Issue 6, 2010, Pages 591-597

Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis

Author keywords

Cystic fibrosis; cystic fibrosis transmembrane conductance regulator; new therapies

Indexed keywords

AGENTS AFFECTING PROTEIN METABOLISM; AMINOGLYCOSIDE DERIVATIVE; ATALUREN; FLAVONOID; GENISTEIN; GENTAMICIN; GLAFANINE; HISTONE DEACETYLASE INHIBITOR; ION CHANNEL; PHOSPHODIESTERASE INHIBITOR; QUERCETIN; SILDENAFIL; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG; VORINOSTAT; VRT 532; VX 770; VX 809;

EID: 77958151782     PISSN: 10705287     EISSN: 15316971     Source Type: Journal    
DOI: 10.1097/MCP.0b013e32833f1d00     Document Type: Review
Times cited : (63)

References (77)
  • 8
    • 0030473619 scopus 로고    scopus 로고
    • Genotype-phenotype correlations in cystic fibrosis
    • DOI 10.1002/(SICI)1099-0496(199612)22:6<387::AID-PPUL7>3.0.CO;2-G
    • Kerem E, Kerem B. Genotype-phenotype correlations in cystic fibrosis. Pediatr Pulmonol 1996; 22:387-395. (Pubitemid 27024442)
    • (1996) Pediatric Pulmonology , vol.22 , Issue.6 , pp. 387-395
    • Kerem, E.1    Kerem, B.2
  • 9
    • 70350045273 scopus 로고    scopus 로고
    • Correctors enhance maturation of DeltaF508 CFTR by promoting interactions between the two halves of the molecule
    • Loo TW, Bartlett MC, Clarke DM. Correctors enhance maturation of DeltaF508 CFTR by promoting interactions between the two halves of the molecule. Biochemistry 2009; 48:9882-9890.
    • (2009) Biochemistry , vol.48 , pp. 9882-9890
    • Loo, T.W.1    Bartlett, M.C.2    Clarke, D.M.3
  • 12
    • 0029616734 scopus 로고
    • Cystic fibrosis: Genotypic and phenotypic variations
    • Zielenski J, Tsui LC. Cystic fibrosis: genotypic and phenotypic variations. Annu Rev Genet 1995; 29:777-807. (Pubitemid 26005359)
    • (1995) Annual Review of Genetics , vol.29 , pp. 777-807
    • Zielenski, J.1    Tsui, L.-C.2
  • 13
    • 0033618404 scopus 로고    scopus 로고
    • C-terminal truncations destabilize the cystic fibrosis transmembrane conductance regulator without impairing its biogenesis: A novel class of mutation
    • Haardt M, Benharouga M, Lechardeur D, et al. C-terminal truncations destabilize the cystic fibrosis transmembrane conductance regulator without impairing its biogenesis: a novel class of mutation. J Biol Chem 1999; 274:21873-21877. (Pubitemid 129519738)
    • (1999) Journal of Biological Chemistry , vol.274 , Issue.31 , pp. 21873-21877
    • Haardt, M.1    Benharouga, M.2    Lechardeur, D.3    Kartner, N.4    Lukacs, G.L.5
  • 14
    • 0027162649 scopus 로고
    • Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis
    • DOI 10.1016/0092-8674(93)90353-R
    • Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell 1993; 73:1251-1254. (Pubitemid 23201140)
    • (1993) Cell , vol.73 , Issue.7 , pp. 1251-1254
    • Welsh, M.J.1    Smith, A.E.2
  • 16
    • 77953120429 scopus 로고    scopus 로고
    • Activation of CFTR by the flavonoid quercetin: Potential use as a biomarker of DF508 CFTR rescue
    • [Epub ahead of print]
    • Pyle LC, Fulton JC, Sloane PA, et al. Activation of CFTR by the flavonoid quercetin: potential use as a biomarker of DF508 CFTR rescue. Am J Respir Cell Mol Biol 2009. [Epub ahead of print]
    • (2009) Am J Respir Cell Mol Biol
    • Pyle, L.C.1    Fulton, J.C.2    Sloane, P.A.3
  • 18
    • 0037020097 scopus 로고    scopus 로고
    • High-affinity activators of cystic fibrosis transmembrane conductance regulator (CFTR) chloride conductance identified by high-throughput screening
    • Ma T, Vetrivel L, Yang H, et al. High-affinity activators of cystic fibrosis transmembrane conductance regulator (CFTR) chloride conductance identified by high-throughput screening. J Biol Chem 2002; 277:37235-37241.
    • (2002) J Biol Chem , vol.277 , pp. 37235-37241
    • Ma, T.1    Vetrivel, L.2    Yang, H.3
  • 20
    • 77958153495 scopus 로고    scopus 로고
    • The conductance assay: A simple assay to measure DF508-CFTR channel activity
    • Abstract 267
    • Thakerar A, Van Driessch A, Bridges RJ. The conductance assay: a simple assay to measure DF508-CFTR channel activity. Ped Pulmonl Suppl 2009; 32:Abstract 267.
    • (2009) Ped Pulmonl Suppl , vol.32
    • Thakerar, A.1    Van Driessch, A.2    Bridges, R.J.3
  • 21
    • 73249114731 scopus 로고    scopus 로고
    • Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
    • Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci U S A 2009; 106:18825-18830.
    • (2009) Proc Natl Acad Sci U S A , vol.106 , pp. 18825-18830
    • Van Goor, F.1    Hadida, S.2    Grootenhuis, P.D.3
  • 22
    • 70349778625 scopus 로고    scopus 로고
    • Problems and opportunities in mutant CFTR rescue
    • Symposium 9.1
    • Galietta LJ. Problems and opportunities in mutant CFTR rescue. Pediatric Pulmonol Suppl 2009; 32: Symposium 9.1.
    • (2009) Pediatric Pulmonol Suppl , vol.32
    • Galietta, L.J.1
  • 23
    • 59849091513 scopus 로고    scopus 로고
    • Direct interaction of a small-molecule modulator with G551D-CFTR, a cystic fibrosis-causing mutation associated with severe disease
    • Pasyk S, Li C, Ramjeesingh M, Bear CE. Direct interaction of a small-molecule modulator with G551D-CFTR, a cystic fibrosis-causing mutation associated with severe disease. Biochem J 2009; 418:185-190.
    • (2009) Biochem J , vol.418 , pp. 185-190
    • Pasyk, S.1    Li, C.2    Ramjeesingh, M.3    Bear, C.E.4
  • 24
    • 66849112041 scopus 로고    scopus 로고
    • Interim results of a phase IIa study of VX-770 to evalaute safety, pharmacokinetics and biomarkers of CFTR activity in cystic fibrosis subjects with G551D
    • Abstract 295
    • Accurso F, Rowe SM, Durie P, et al. Interim results of a phase IIa study of VX-770 to evalaute safety, pharmacokinetics and biomarkers of CFTR activity in cystic fibrosis subjects with G551D. Pediatr Pulmonol 2008; 43:Abstract 295.
    • (2008) Pediatr Pulmonol , vol.43
    • Accurso, F.1    Rowe, S.M.2    Durie, P.3
  • 25
    • 77952574720 scopus 로고    scopus 로고
    • NPD evaluation of ion transport in G551D CF patients treated with a CFTR potentiator
    • Abstract 222
    • Clancy JP, Rowe SM, Durie PR, et al. NPD evaluation of ion transport in G551D CF patients treated with a CFTR potentiator. Ped Pulmonol Suppl 2009; 32: Abstract 222.
    • (2009) Ped Pulmonol Suppl , vol.32
    • Clancy, J.P.1    Rowe, S.M.2    Durie, P.R.3
  • 26
    • 77958152352 scopus 로고    scopus 로고
    • Effect of VX-770, a CFTR potentiator, on spirometry and QOL assessment in subjects with CF and the G551D-CFTR mutation
    • Abstract 217
    • Boyle M, Clancy JP, Rowe SM, et al. Effect of VX-770, a CFTR potentiator, on spirometry and QOL assessment in subjects with CF and the G551D-CFTR mutation. Ped Pulmonol Supp 2009; 32: Abstract 217.
    • (2009) Ped Pulmonol Supp , vol.32
    • Boyle, M.1    Clancy, J.P.2    Rowe, S.M.3
  • 27
    • 0030809817 scopus 로고    scopus 로고
    • In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR
    • Rubenstein RC, Egan ME, Zeitlin PL. In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR. J Clin Invest 1997; 100:2457-2465.
    • (1997) J Clin Invest , vol.100 , pp. 2457-2465
    • Rubenstein, R.C.1    Egan, M.E.2    Zeitlin, P.L.3
  • 28
    • 0031889082 scopus 로고    scopus 로고
    • A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: Partial restoration of nasal epithelial CFTR function
    • Rubenstein RC, Zeitlin PL. A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function. Am J Respir Crit Care Med 1998; 157:484-490.
    • (1998) Am J Respir Crit Care Med , vol.157 , pp. 484-490
    • Rubenstein, R.C.1    Zeitlin, P.L.2
  • 31
    • 41149113942 scopus 로고    scopus 로고
    • Enhanced cell-surface stability of rescued ΔF508 cystic fibrosis transmembrane conductance regulator (CFTR) by pharmacological chaperones
    • DOI 10.1042/BJ20071420
    • Varga K, Goldstein RF, Jurkuvenaite A, et al. Enhanced cell-surface stability of rescued DeltaF508 cystic fibrosis transmembrane conductance regulator (CFTR) by pharmacological chaperones. Biochem J 2008; 410:555-564. (Pubitemid 351429026)
    • (2008) Biochemical Journal , vol.410 , Issue.3 , pp. 555-564
    • Varga, K.1    Goldstein, R.F.2    Jurkuvenaite, A.3    Chen, L.4    Matalon, S.5    Sorscher, E.J.6    Bebok, Z.7    Collawn, J.F.8
  • 32
    • 69249227507 scopus 로고    scopus 로고
    • Dynasore inhibits removal of wild-type and DeltaF508 cystic fibrosis transmembrane conductance regulator (CFTR) from the plasma membrane
    • Young A, Gentzsch M, Abban CY, et al. Dynasore inhibits removal of wild-type and DeltaF508 cystic fibrosis transmembrane conductance regulator (CFTR) from the plasma membrane. Biochem J 2009; 421:377-385.
    • (2009) Biochem J , vol.421 , pp. 377-385
    • Young, A.1    Gentzsch, M.2    Abban, C.Y.3
  • 33
    • 77956237958 scopus 로고    scopus 로고
    • A chemical corrector modifies the channel function of F508del-CFTR
    • Kim Chiaw P, Wellhauser L, Huan LJ, et al. A chemical corrector modifies the channel function of F508del-CFTR. Mol Pharmacol 2010; 78:411-418.
    • (2010) Mol Pharmacol , vol.78 , pp. 411-418
    • Kim Chiaw, P.1    Wellhauser, L.2    Huan, L.J.3
  • 34
    • 66849129301 scopus 로고    scopus 로고
    • A small-molecule modulator interacts directly with deltaPhe508-CFTR to modify its ATPase activity and conformational stability
    • Wellhauser L, Kim Chiaw P, Pasyk S, et al. A small-molecule modulator interacts directly with deltaPhe508-CFTR to modify its ATPase activity and conformational stability. Mol Pharmacol 2009; 75:1430-1438.
    • (2009) Mol Pharmacol , vol.75 , pp. 1430-1438
    • Wellhauser, L.1    Kim Chiaw, P.2    Pasyk, S.3
  • 35
    • 77958150820 scopus 로고    scopus 로고
    • Press release, Vertex Pharmaceuticals, Inc. [Accessed 2 August 2010]
    • Press release, Vertex Pharmaceuticals, Inc. http://investors.vrtx.com/ releasedetail. cfm?releaseid=442429. [Accessed 2 August 2010]
  • 36
    • 70349778630 scopus 로고    scopus 로고
    • VX-809, a CFTR corrector, increases the cell surface density of functional F508del-CFTR in preclinical models of cystic fibrosis
    • Abstract 154
    • Van Goor F, Haditha S, Grootenhuis PD, et al. VX-809, a CFTR corrector, increases the cell surface density of functional F508del-CFTR in preclinical models of cystic fibrosis. Ped Pulmonol Supp 2009; 32:Abstract 154.
    • (2009) Ped Pulmonol Supp , vol.32
    • Van Goor, F.1    Haditha, S.2    Grootenhuis, P.D.3
  • 39
    • 77958150930 scopus 로고    scopus 로고
    • Inhaled PDE5 inhibitors restore chloride transport in cystic fibrosis mice
    • [Epub ahead of print]
    • Lubamba B, Lebacq J, Reychler G, et al. Inhaled PDE5 inhibitors restore chloride transport in cystic fibrosis mice. Eur Respir J 2010. [Epub ahead of print]
    • (2010) Eur Respir J
    • Lubamba, B.1    Lebacq, J.2    Reychler, G.3
  • 40
    • 77952399647 scopus 로고    scopus 로고
    • Correction of the Delta phe508 cystic fibrosis transmembrane conductance regulator trafficking defect by the bioavailable compound glafenine
    • Robert R, Carlile GW, Liao J, et al. Correction of the Delta phe508 cystic fibrosis transmembrane conductance regulator trafficking defect by the bioavailable compound glafenine. Mol Pharmacol 2010; 77:922-930.
    • (2010) Mol Pharmacol , vol.77 , pp. 922-930
    • Robert, R.1    Carlile, G.W.2    Liao, J.3
  • 42
    • 65449160927 scopus 로고    scopus 로고
    • Inhibition of histone deacetylases: A pharmacological approach to the treatment of noncancer disorders
    • Wiech NL, Fisher JF, Helquist P, Wiest O. Inhibition of histone deacetylases: a pharmacological approach to the treatment of noncancer disorders. Curr Top Med Chem 2009; 9:257-271.
    • (2009) Curr Top Med Chem , vol.9 , pp. 257-271
    • Wiech, N.L.1    Fisher, J.F.2    Helquist, P.3    Wiest, O.4
  • 43
    • 77950428804 scopus 로고    scopus 로고
    • Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis
    • Hutt DM, Herman D, Rodrigues AP, et al. Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis. Nat Chem Biol 2010; 6:25-33.
    • (2010) Nat Chem Biol , vol.6 , pp. 25-33
    • Hutt, D.M.1    Herman, D.2    Rodrigues, A.P.3
  • 44
    • 50249175120 scopus 로고    scopus 로고
    • Chemical and biological approaches synergize to ameliorate protein-folding diseases
    • Mu TW, Ong DS, Wang YJ, et al. Chemical and biological approaches synergize to ameliorate protein-folding diseases. Cell 2008; 134:769-781.
    • (2008) Cell , vol.134 , pp. 769-781
    • Mu, T.W.1    Ong, D.S.2    Wang, Y.J.3
  • 45
    • 34547093657 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics: Old drugs and new therapeutic approaches
    • DOI 10.1007/s00018-007-7034-x
    • Hermann T. Aminoglycoside antibiotics: old drugs and new therapeutic approaches. Cell Mol Life Sci 2007; 64:1841-1852. (Pubitemid 47096588)
    • (2007) Cellular and Molecular Life Sciences , vol.64 , Issue.14 , pp. 1841-1852
    • Hermann, T.1
  • 46
    • 0035253591 scopus 로고    scopus 로고
    • Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-Liduronidase activity and reduces lysosomal glycosaminoglycan accumulation
    • Keeling KM, Brooks DA, Hopwood JJ, et al. Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-Liduronidase activity and reduces lysosomal glycosaminoglycan accumulation. Hum Mol Genet 2001; 10:291-299.
    • (2001) Hum Mol Genet , vol.10 , pp. 291-299
    • Keeling, K.M.1    Brooks, D.A.2    Hopwood, J.J.3
  • 47
    • 0029994529 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations
    • DOI 10.1038/nm0496-467
    • Howard M, Frizzell RA, Bedwell DM. Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations. Nat Med 1996; 2:467-469. (Pubitemid 26191920)
    • (1996) Nature Medicine , vol.2 , Issue.4 , pp. 467-469
    • Howard, M.1    Frizzell, R.A.2    Bedwell, D.M.3
  • 49
    • 0038811889 scopus 로고    scopus 로고
    • Aminoglycoside and its derivatives as ligands to target the ribosome
    • Tok JB, Bi L. Aminoglycoside and its derivatives as ligands to target the ribosome. Curr Top Med Chem 2003; 3:1001-1019.
    • (2003) Curr Top Med Chem , vol.3 , pp. 1001-1019
    • Tok, J.B.1    Bi, L.2
  • 50
    • 0029153552 scopus 로고
    • The efficiency of translation termination is determined by a synergistic interplay between upstream and downstream sequences in Saccharomyces cerevisiae
    • Bonetti B, Fu L, Moon J, Bedwell DM. The efficiency of translation termination is determined by a synergistic interplay between upstream and downstream sequences in Saccharomyces cerevisiae. J Mol Biol 1995; 251:334-345.
    • (1995) J Mol Biol , vol.251 , pp. 334-345
    • Bonetti, B.1    Fu, L.2    Moon, J.3    Bedwell, D.M.4
  • 52
    • 0033929810 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system
    • DOI 10.1017/S1355838200000716
    • Manuvakhova M, Keeling K, Bedwell DM. Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system. RNA 2000; 6:1044-1055. (Pubitemid 30471140)
    • (2000) RNA , vol.6 , Issue.7 , pp. 1044-1055
    • Manuvakhova, M.1    Keeling, K.2    Bedwell, D.M.3
  • 57
    • 77958150460 scopus 로고    scopus 로고
    • Ataluren (PTC124) Induces CFTR Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis
    • [Epub ahead of print]
    • Sermet-Gaudelus I, De Boeck K, Casimir GJ, et al. Ataluren (PTC124) Induces CFTR Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis. Am J Respir Crit Care Med 2010. [Epub ahead of print]
    • (2010) Am J Respir Crit Care Med
    • Sermet-Gaudelus, I.1    De Boeck, K.2    Casimir, G.J.3
  • 58
    • 0036351425 scopus 로고    scopus 로고
    • Expression of CTNS alleles: Subcellular localization and aminoglycoside correction in Vitro
    • DOI 10.1006/mgme.2001.3272
    • Helip-Wooley A, Park MA, Lemons RM, Thoene JG. Expression of CTNS alleles: subcellular localization and aminoglycoside correction in vitro. Mol Genet Metab 2002; 75:128-133. (Pubitemid 34971605)
    • (2002) Molecular Genetics and Metabolism , vol.75 , Issue.2 , pp. 128-133
    • Helip-Wooley, A.1    Park, M.A.2    Lemons, R.M.3    Thoene, J.G.4
  • 59
    • 0034923247 scopus 로고    scopus 로고
    • Aminoglycoside-mediated suppression of nonsense mutations in late infantile neuronal ceroid lipofuscinosis
    • DOI 10.1053/ejpn.2001.0466
    • Sleat DE, Sohar I, Gin RM, Lobel P. Aminoglycoside-mediated suppression of nonsense mutations in late infantile neuronal ceroid lipofuscinosis. Eur J Paediatr Neurol 2001; 5 (Suppl A:):57-62. (Pubitemid 32677113)
    • (2001) European Journal of Paediatric Neurology , vol.5 , Issue.SUPPL. A , pp. 57-62
    • Sleat, D.E.1    Sohar, I.2    Gin, R.M.3    Lobel, P.4
  • 60
    • 0035997219 scopus 로고    scopus 로고
    • Clinically relevant aminoglycosides can suppress disease-associated premature stop mutations in the IDUA and P53 cDNAs in a mammalian translation system
    • Keeling KM, Bedwell DM. Clinically relevant aminoglycosides can suppress disease-associated premature stop mutations in the IDUA and P53 cDNAs in a mammalian translation system. J Mol Med 2002; 80:367-376.
    • (2002) J Mol Med , vol.80 , pp. 367-376
    • Keeling, K.M.1    Bedwell, D.M.2
  • 62
    • 0036379141 scopus 로고    scopus 로고
    • Aminoglycoside suppression of a premature stop mutation in a Cftr-/-mouse carrying a human CFTR-G542X transgene
    • Du M, Jones JR, Lanier J, et al. Aminoglycoside suppression of a premature stop mutation in a Cftr-/-mouse carrying a human CFTR-G542X transgene. J Mol Med 2002; 80:595-604.
    • (2002) J Mol Med , vol.80 , pp. 595-604
    • Du, M.1    Jones, J.R.2    Lanier, J.3
  • 63
    • 33745628041 scopus 로고    scopus 로고
    • Clinical doses of amikacin provide more effective suppression of the human CFTR-G542X stop mutation than gentamicin in a transgenic CF mouse model
    • DOI 10.1007/s00109-006-0045-5
    • Du M, Keeling KM, Fan L, et al. Clinical doses of amikacin provide more effective suppression of the human CFTR-G542X stop mutation than gentamicin in a transgenic CF mouse model. J Mol Med 2006; 84:573-582. (Pubitemid 43992849)
    • (2006) Journal of Molecular Medicine , vol.84 , Issue.7 , pp. 573-582
    • Du, M.1    Keeling, K.M.2    Fan, L.3    Liu, X.4    Kovacs, T.5    Sorscher, E.6    Bedwell, D.M.7
  • 65
    • 65649136885 scopus 로고    scopus 로고
    • Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations
    • Nudelman I, Rebibo-Sabbah A, Cherniavsky M, et al. Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations. J Med Chem 2009; 52:2836-2845.
    • (2009) J Med Chem , vol.52 , pp. 2836-2845
    • Nudelman, I.1    Rebibo-Sabbah, A.2    Cherniavsky, M.3
  • 66
    • 77958154218 scopus 로고    scopus 로고
    • Enhanced activity for translational readthrough and reduced toxicity demonstrated by the synthetic aminoglycoside NB54
    • Abstract 218
    • Rowe SM, Backer K, Tang L, et al. Enhanced activity for translational readthrough and reduced toxicity demonstrated by the synthetic aminoglycoside NB54. Ped Pulmonol Supp 2009; 32:Abstract 218.
    • (2009) Ped Pulmonol Supp , vol.32
    • Rowe, S.M.1    Backer, K.2    Tang, L.3
  • 70
    • 50149098401 scopus 로고    scopus 로고
    • Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: A prospective phase II trial
    • Kerem E, Hirawat S, Armoni S, et al. Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. Lancet 2008; 372:719-727.
    • (2008) Lancet , vol.372 , pp. 719-727
    • Kerem, E.1    Hirawat, S.2    Armoni, S.3
  • 71
    • 34347219633 scopus 로고    scopus 로고
    • A phase II study of PTC124 in CF patients harboring premature stop mutations
    • Abstract 269
    • Clancy JP, Konstan MW, Rowe SM, et al. A phase II study of PTC124 in CF patients harboring premature stop mutations. Ped Pulmonol Suppl 2006; 41:Abstract 269.
    • (2006) Ped Pulmonol Suppl , vol.41
    • Clancy, J.P.1    Konstan, M.W.2    Rowe, S.M.3
  • 72
    • 77957827856 scopus 로고    scopus 로고
    • An international randomized multicenter comparison of nasal potential difference techniques
    • [Epub ahead of print]
    • SolomonGM, Konstan MW, WilschanskiM, et al. An international randomized multicenter comparison of nasal potential difference techniques. Chest 2010. [Epub ahead of print]
    • (2010) Chest
    • Solomon, G.M.1    Konstan, M.W.2    Wilschanski, M.3
  • 75
    • 62549134976 scopus 로고    scopus 로고
    • Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression
    • Auld DS, Thorne N, Maguire WF, Inglese J. Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression. Proc Natl Acad Sci U S A 2009; 106:3585-3590.
    • (2009) Proc Natl Acad Sci U S A , vol.106 , pp. 3585-3590
    • Auld, D.S.1    Thorne, N.2    Maguire, W.F.3    Inglese, J.4
  • 76
    • 77950430317 scopus 로고    scopus 로고
    • Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124
    • Auld DS, Lovell S, Thorne N, et al. Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124. Proc Natl Acad Sci U S A 2010; 107:4878-4883.
    • (2010) Proc Natl Acad Sci U S A , vol.107 , pp. 4878-4883
    • Auld, D.S.1    Lovell, S.2    Thorne, N.3
  • 77
    • 67349124721 scopus 로고    scopus 로고
    • PTC124 treatment over 3 months improves pharmacodynamic and clinical parameters in patients with nonsense-mutation-mediated CF
    • Symposium 22
    • Wilschanski M, Armoni S, Yaacov Y, et al. PTC124 treatment over 3 months improves pharmacodynamic and clinical parameters in patients with nonsense-mutation-mediated CF. J Cyst Fibros 2008; 7:Symposium 22
    • (2008) J Cyst Fibros , vol.7
    • Wilschanski, M.1    Armoni, S.2    Yaacov, Y.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.