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Volumn 115, Issue 9, 2015, Pages 546-555

Cystic fibrosis: A novel pharmacologic approach to cystic fibrosis transmembrane regulator modulation therapy

Author keywords

[No Author keywords available]

Indexed keywords

1,3 BENZODIOXOLE DERIVATIVE; AMINOPHENOL DERIVATIVE; AMINOPYRIDINE DERIVATIVE; ATALUREN; CFTR PROTEIN, HUMAN; CHLORIDE CHANNEL STIMULATING AGENT; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DNA; IVACAFTOR; LUMACAFTOR; OXADIAZOLE DERIVATIVE; QUINOLONE DERIVATIVE;

EID: 84956496199     PISSN: 00986151     EISSN: 19451997     Source Type: Journal    
DOI: 10.7556/jaoa.2015.112     Document Type: Review
Times cited : (3)

References (37)
  • 1
    • 84956465171 scopus 로고    scopus 로고
    • Accessed July 30, 2015
    • Cystic Fibrosis Foundation. Patient registry annual data report 2013. https://www.cff.org/2013_CFF_Patient_Registry_Annual_Data_Report.pdf. Accessed July 30, 2015.
    • Patient Registry Annual Data Report 2013
  • 2
    • 79960150961 scopus 로고    scopus 로고
    • New and investigational treatments in cystic fibrosis
    • Narasimhan M, Cohen R. New and investigational treatments in cystic fibrosis. Ther Adv Respir Dis. 2011;5(4):275-282.
    • (2011) Ther Adv Respir Dis , vol.5 , Issue.4 , pp. 275-282
    • Narasimhan, M.1    Cohen, R.2
  • 3
    • 80455162465 scopus 로고    scopus 로고
    • A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
    • Ramsey B, Davies J, McElvaney N, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672.
    • (2011) N Engl J Med , vol.365 , Issue.18 , pp. 1663-1672
    • Ramsey, B.1    Davies, J.2    McElvaney, N.3
  • 4
    • 0036801803 scopus 로고    scopus 로고
    • Demographics of the UK cystic fibrosis population: Implications for neonatal screening
    • McCormick J, Green MW, Mehta G, et al. Demographics of the UK cystic fibrosis population: implications for neonatal screening. Eur J Hum Genet. 2002;10(10):583-590.
    • (2002) Eur J Hum Genet , vol.10 , Issue.10 , pp. 583-590
    • McCormick, J.1    Green, M.W.2    Mehta, G.3
  • 5
    • 33845355490 scopus 로고    scopus 로고
    • Cystic fibrosis in adults: An overview for the internist
    • Gershman AJ, Mehta AC, Infeld M, Budey MM. Cystic fibrosis in adults: an overview for the internist. Cleve Clin J Med. 2006;73(12):1065-1074.
    • (2006) Cleve Clin J Med , vol.73 , Issue.12 , pp. 1065-1074
    • Gershman, A.J.1    Mehta, A.C.2    Infeld, M.3    Budey, M.M.4
  • 6
    • 20144389008 scopus 로고    scopus 로고
    • Guide to bone health and disease in cystic fibrosis
    • Aris RM, Merkel PA, Bachrach LK, et al. Guide to bone health and disease in cystic fibrosis. J Clin Endocrinol Metab. 2005;90(3):1888-1896.
    • (2005) J Clin Endocrinol Metab , vol.90 , Issue.3 , pp. 1888-1896
    • Aris, R.M.1    Merkel, P.A.2    Bachrach, L.K.3
  • 9
    • 0027162649 scopus 로고
    • Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis
    • Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell. 1993;73(7):1251-1254.
    • (1993) Cell , vol.73 , Issue.7 , pp. 1251-1254
    • Welsh, M.J.1    Smith, A.E.2
  • 10
    • 84864757410 scopus 로고    scopus 로고
    • Cystic fibrosis transmembrane conductance regulator-modifying medications: The future of cystic fibrosis treatment. [published online June 26, 2012]
    • Pettits R. Cystic fibrosis transmembrane conductance regulator-modifying medications: the future of cystic fibrosis treatment. [published online June 26, 2012] Ann Pharmacother. 2012;46(7-8):1065-1074. doi:10.1345/aph1.R076.
    • (2012) Ann Pharmacother. , vol.46 , Issue.7-8 , pp. 1065-1074
    • Pettits, R.1
  • 11
    • 84866295520 scopus 로고    scopus 로고
    • Cystic fibrosis: Insight into CFTR pathophysiology and pharmacotherapy
    • Lubamba B, Dhooghe B, Noel S, et al. Cystic fibrosis: insight into CFTR pathophysiology and pharmacotherapy. Clin Biochem. 2012;45(15):1132-1144.
    • (2012) Clin Biochem , vol.45 , Issue.15 , pp. 1132-1144
    • Lubamba, B.1    Dhooghe, B.2    Noel, S.3
  • 12
    • 0141863491 scopus 로고    scopus 로고
    • Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations
    • Wilschanski M, Yahav Y, Yaacov Y, et al. Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations. N Engl J Med. 2003;349(15):1433-1441.
    • (2003) N Engl J Med , vol.349 , Issue.15 , pp. 1433-1441
    • Wilschanski, M.1    Yahav, Y.2    Yaacov, Y.3
  • 13
    • 84886430218 scopus 로고    scopus 로고
    • Optimization of anti-pseudomonal antibiotics for cystic fibrosis pulmonary exacerbations
    • Young DC, Zobell JT, Stockmann C, et al. Optimization of anti-pseudomonal antibiotics for cystic fibrosis pulmonary exacerbations. Pediatr Pulmonol. 2013;48(11):1047-1061.
    • (2013) Pediatr Pulmonol , vol.48 , Issue.11 , pp. 1047-1061
    • Young, D.C.1    Zobell, J.T.2    Stockmann, C.3
  • 14
    • 0029994529 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics restore CFTR function by overcoming premature stop codons
    • Howard M, Frizzell RA, Bedwell DM. Aminoglycoside antibiotics restore CFTR function by overcoming premature stop codons. Nat Med. 1996;2(4):467-469.
    • (1996) Nat Med , vol.2 , Issue.4 , pp. 467-469
    • Howard, M.1    Frizzell, R.A.2    Bedwell, D.M.3
  • 15
    • 0034961464 scopus 로고    scopus 로고
    • Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis
    • Clancy JP, Bebok Z, Ruiz F, et al. Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis. Am J Respir Crit Care Med. 2001;163(7):1683-1692.
    • (2001) Am J Respir Crit Care Med , vol.163 , Issue.7 , pp. 1683-1692
    • Clancy, J.P.1    Bebok, Z.2    Ruiz, F.3
  • 16
    • 34547093657 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics: Old drugs and new therapeutic approaches
    • Hermann T. Aminoglycoside antibiotics: old drugs and new therapeutic approaches. Cell Mol Life Sci. 2007;64(14):1841-1852.
    • (2007) Cell Mol Life Sci , vol.64 , Issue.14 , pp. 1841-1852
    • Hermann, T.1
  • 17
    • 34247200483 scopus 로고    scopus 로고
    • In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: A pilot study
    • Sermet-Gaudelus I, Renouil M, Fajac A, et al. In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study. BMC Med. 2007;5:5.
    • (2007) BMC Med , vol.5 , pp. 5
    • Sermet-Gaudelus, I.1    Renouil, M.2    Fajac, A.3
  • 18
    • 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(9640):719-727.
    • (2008) Lancet , vol.372 , Issue.9640 , pp. 719-727
    • Kerem, E.1    Hirawat, S.2    Armoni, S.3
  • 20
    • 79960187095 scopus 로고    scopus 로고
    • Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis
    • Wilschanski M, Miller LL, Shoseyov D, et al. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur Respir J. 2011;38(1):59-69.
    • (2011) Eur Respir J , vol.38 , Issue.1 , pp. 59-69
    • Wilschanski, M.1    Miller, L.L.2    Shoseyov, D.3
  • 21
    • 84910627623 scopus 로고    scopus 로고
    • Ataluren: First global approval
    • Ryan N J. Ataluren: first global approval. Drugs. 2014;74(14):1709-1714.
    • (2014) Drugs , vol.74 , Issue.14 , pp. 1709-1714
    • Ryan, N.J.1
  • 22
    • 84904410000 scopus 로고    scopus 로고
    • CFTR modulators for the treatment of cystic fibrosis
    • Pettit RS, Fellner C. CFTR modulators for the treatment of cystic fibrosis. PT. 2014;39(7):500-511.
    • (2014) PT , vol.39 , Issue.7 , pp. 500-511
    • Pettit, R.S.1    Fellner, C.2
  • 23
    • 81755163563 scopus 로고    scopus 로고
    • Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
    • Van Goor F, Hadida S, Grootenhuis PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci U S A. 2011;180(46):18843-18848.
    • (2011) Proc Natl Acad Sci U S A , vol.180 , Issue.46 , pp. 18843-18848
    • Van Goor, F.1    Hadida, S.2    Grootenhuis, P.D.3
  • 24
    • 84859793396 scopus 로고    scopus 로고
    • VX-809, an investigational CFTR corrector, in combination with VX-770, an investigational CFTR potentiator, in subjects with CF and homozygous for the F508del-CFTR mutation [abstract 212]
    • Boyle M, Bell S, Konstan MW, et al. VX-809, an investigational CFTR corrector, in combination with VX-770, an investigational CFTR potentiator, in subjects with CF and homozygous for the F508del-CFTR mutation [abstract 212]. Pediatr Pulmonol. 2011;S34(suppl):287. doi:10.1002/ppul.21583.
    • (2011) Pediatr Pulmonol , vol.34 , pp. 287
    • Boyle, M.1    Bell, S.2    Konstan, M.W.3
  • 25
    • 84855202429 scopus 로고    scopus 로고
    • Results of 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, et al. Results of phase IIa study of VX-809, an investigational CFTR corrector compound in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax. 2012;67(1):12-18.
    • (2012) Thorax , vol.67 , Issue.1 , pp. 12-18
    • Clancy, J.P.1    Rowe, S.M.2    Accurso, F.J.3
  • 27
    • 80455162465 scopus 로고    scopus 로고
    • A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
    • Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672.
    • (2011) N Engl J Med , vol.365 , Issue.18 , pp. 1663-1672
    • Ramsey, B.W.1    Davies, J.2    McElvaney, N.G.3
  • 28
    • 84865853894 scopus 로고    scopus 로고
    • Ivacaftor in subjects with cystic fibrosis who are homozygous for F508del-CFTR mutation
    • Flume PA, Liou TG, Borowitz DS, et al. Ivacaftor in subjects with cystic fibrosis who are homozygous for F508del-CFTR mutation. Chest. 2012;142(3):718-724.
    • (2012) Chest , vol.142 , Issue.3 , pp. 718-724
    • Flume, P.A.1    Liou, T.G.2    Borowitz, D.S.3
  • 29
    • 84956555346 scopus 로고    scopus 로고
    • Boston, MA: Vertex Pharmaceuticals Inc, Accessed September 29, 2014
    • KALYDECO (ivacaftor) Tablets, for oral use [package insert]. Boston, MA: Vertex Pharmaceuticals Inc; 2014. http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/203188s004lbl.pdf. Accessed September 29, 2014.
    • (2014) Tablets, for Oral Use [Package Insert]
  • 30
    • 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 Fibrosis. 2012;11(3):237-245.
    • (2012) J Cyst Fibrosis , vol.11 , Issue.3 , pp. 237-245
    • Yu, H.1    Burton, B.2    Huang, C.J.3
  • 31
    • 84904410000 scopus 로고    scopus 로고
    • CFTR modulators for the treatment of cystic fibrosis
    • Pettit R, Fellner C. CFTR modulators for the treatment of cystic fibrosis. P T. 2014;39(7):500-511.
    • (2014) P T , vol.39 , Issue.7 , pp. 500-511
    • Pettit, R.1    Fellner, C.2
  • 32
    • 84937035647 scopus 로고    scopus 로고
    • Lumacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR
    • Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Eng J Med. 2015;373:220-231. doi:10.1056/NEJMoa1409547.
    • (2015) N Eng J Med , vol.373 , pp. 220-231
    • Wainwright, C.E.1    Elborn, J.S.2    Ramsey, B.W.3
  • 34
    • 84901398808 scopus 로고    scopus 로고
    • VX-809 and related corrector compounds exhibit secondary activity stabilizing active F508del-CFTR after its partial rescue to the cell surface
    • Eckford PDW, Ramjeesigh M, Molinski S, et al. VX-809 and related corrector compounds exhibit secondary activity stabilizing active F508del-CFTR after its partial rescue to the cell surface. Chem Biol. 2014;21(5):666-678.
    • (2014) Chem Biol , vol.21 , Issue.5 , pp. 666-678
    • Eckford, P.1    Ramjeesigh, M.2    Molinski, S.3
  • 37
    • 0036120193 scopus 로고    scopus 로고
    • The cystic fibrosis transmembrane conductance regulator: An intiriguing protein with pleiotropic functions
    • Vankeerberghen A, Cuppens H, Cassiman JJ. The cystic fibrosis transmembrane conductance regulator: an intiriguing protein with pleiotropic functions. J Cyst Fibros. 2002;1(1):13-29.
    • (2002) J Cyst Fibros , vol.1 , Issue.1 , pp. 13-29
    • Vankeerberghen, A.1    Cuppens, H.2    Cassiman, J.J.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.