메뉴 건너뛰기




Volumn 51, Issue 3, 2014, Pages 354-362

A genomic signature approach to rescue ΔF508-cystic fibrosis transmembrane conductance regulator biosynthesis and function

Author keywords

Airway epithelia; Connectivity map; Corrector compound 18; Cystic fibrosis; Small molecule compound

Indexed keywords

ANION CHANNEL; BIPERIDEN; CFTR CORRECTOR COMPOUND 18; CHLORIDE CHANNEL AFFECTING AGENT; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; MICRORNA; MICRORNA 138; MIDODRINE; NEOSTIGMINE; PIZOTIFEN; PYRIDOSTIGMINE; UNCLASSIFIED DRUG; VALPROIC ACID;

EID: 85047689539     PISSN: 10441549     EISSN: 15354989     Source Type: Journal    
DOI: 10.1165/rcmb.2014-0007OC     Document Type: Article
Times cited : (12)

References (47)
  • 1
    • 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. Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive. Nature 1992;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
  • 2
    • 0030154620 scopus 로고    scopus 로고
    • Chemical chaperones correct the mutant phenotype of the DeltaF508 cystic fibrosis transmembrane conductance regulator protein
    • Brown CR, Hong-Brown LQ, Biwersi J, Verkman AS, Welch WJ. Chemical chaperones correct the mutant phenotype of the delta F508 cystic fibrosis transmembrane conductance regulator protein. Cell Stress Chaperones 1996;1:117-125. (Pubitemid 126670773)
    • (1996) Cell Stress and Chaperones , vol.1 , Issue.2 , pp. 117-125
    • Brown, C.R.1    Hong-Brown, L.Q.2    Biwersi, J.3    Verkman, A.S.4    Welch, W.J.5
  • 7
    • 80053198436 scopus 로고    scopus 로고
    • Rescue of the mutant CFTR chloride channel by pharmacological correctors and low temperature analyzed by gene expression profiling
    • Sondo E, Tomati V, Caci E, Esposito AI, Pfeffer U, Pedemonte N, Galietta LJ. Rescue of the mutant CFTR chloride channel by pharmacological correctors and low temperature analyzed by gene expression profiling. Am J Physiol Cell Physiol 2011;301:C872-C885.
    • (2011) Am J Physiol Cell Physiol , vol.301
    • Sondo, E.1    Tomati, V.2    Caci, E.3    Esposito, A.I.4    Pfeffer, U.5    Pedemonte, N.6    Galietta, L.J.7
  • 11
  • 16
    • 84872074928 scopus 로고    scopus 로고
    • Gene expression-based chemical genomics identifies heat-shock protein 90 inhibitors as potential therapeutic drugs in cholangiocarcinoma
    • Chen MH, Lin KJ, Yang WL, Kao YW, Chen TW, Chao SC, Chang PM, Liu CY, Tzeng CH, Chao Y, et al. Gene expression-based chemical genomics identifies heat-shock protein 90 inhibitors as potential therapeutic drugs in cholangiocarcinoma. Cancer 2013;119:293-303.
    • (2013) Cancer , vol.119 , pp. 293-303
    • Chen, M.H.1    Lin, K.J.2    Yang, W.L.3    Kao, Y.W.4    Chen, T.W.5    Chao, S.C.6    Chang, P.M.7    Liu, C.Y.8    Tzeng, C.H.9    Chao, Y.10
  • 17
    • 84866070620 scopus 로고    scopus 로고
    • Comparative gene expression profiling of benign and malignant lesions reveals candidate therapeutic compounds for leiomyosarcoma
    • Edris B, Fletcher JA, West RB, van de Rijn M, Beck AH. Comparative gene expression profiling of benign and malignant lesions reveals candidate therapeutic compounds for leiomyosarcoma. Sarcoma 2012;2012:805614.
    • (2012) Sarcoma , vol.2012 , pp. 805614
    • Edris, B.1    Fletcher, J.A.2    West, R.B.3    Van De Rijn, M.4    Beck, A.H.5
  • 21
    • 0035937847 scopus 로고    scopus 로고
    • Conformational and temperature-sensitive stability defects of the delta F508 cystic fibrosis transmembrane conductance regulator in postendoplasmic reticulum compartments
    • Sharma M, Benharouga M, Hu W, Lukacs GL. Conformational and temperature-sensitive stability defects of the delta F508 cystic fibrosis transmembrane conductance regulator in postendoplasmic reticulum compartments. J Biol Chem 2001;276:8942-8950.
    • (2001) J Biol Chem , vol.276 , pp. 8942-8950
    • Sharma, M.1    Benharouga, M.2    Hu, W.3    Lukacs, G.L.4
  • 23
    • 4444371518 scopus 로고    scopus 로고
    • Modulation of DeltaF508 cystic fibrosis transmembrane regulator trafficking and function with 4-phenylbutyrate and flavonoids
    • DOI 10.1165/rcmb.2002-0086OC
    • Lim M, McKenzie K, Floyd AD, Kwon E, Zeitlin PL. Modulation of deltaF508 cystic fibrosis transmembrane regulator trafficking and function with 4-phenylbutyrate and flavonoids. Am J Respir Cell Mol Biol 2004;31:351-357. (Pubitemid 39180306)
    • (2004) American Journal of Respiratory Cell and Molecular Biology , vol.31 , Issue.3 , pp. 351-357
    • Lim, M.1    McKenzie, K.2    Floyd, A.D.3    Kwon, E.4    Zeitlin, P.L.5
  • 24
    • 84870735685 scopus 로고    scopus 로고
    • Pharmacological correctors of mutant CFTR mistrafficking
    • Pedemonte N, Galietta LJ. Pharmacological correctors of mutant CFTR mistrafficking. Front Pharmacol 2012;3:175.
    • (2012) Front Pharmacol , vol.3 , pp. 175
    • Pedemonte, N.1    Galietta, L.J.2
  • 26
    • 84885670457 scopus 로고    scopus 로고
    • Managing the underlying cause of cystic fibrosis: A future role for potentiators and correctors
    • Galietta LJ. Managing the underlying cause of cystic fibrosis: a future role for potentiators and correctors. Paediatr Drugs 2013;15:393-402.
    • (2013) Paediatr Drugs , vol.15 , pp. 393-402
    • Galietta, L.J.1
  • 31
    • 80053179441 scopus 로고    scopus 로고
    • Cyanoquinolines with independent corrector and potentiator activities restore DeltaPhe508-cystic fibrosis transmembrane conductance regulator chloride channel function in cystic fibrosis
    • Phuan PW, Yang B, Knapp JM, Wood AB, Lukacs GL, Kurth MJ, Verkman AS. Cyanoquinolines with independent corrector and potentiator activities restore DeltaPhe508-cystic fibrosis transmembrane conductance regulator chloride channel function in cystic fibrosis. Mol Pharmacol 2011;80:683-693.
    • (2011) Mol Pharmacol , vol.80 , pp. 683-693
    • Phuan, P.W.1    Yang, B.2    Knapp, J.M.3    Wood, A.B.4    Lukacs, G.L.5    Kurth, M.J.6    Verkman, A.S.7
  • 34
    • 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. 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 2012;67:12-18.
    • (2012) 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
  • 35
    • 82955233645 scopus 로고    scopus 로고
    • Uptake, efficacy, and systemic distribution of naked, inhaled short interfering RNA (siRNA) and locked nucleic acid (LNA) antisense
    • Moschos SA, Frick M, Taylor B, Turnpenny P, Graves H, Spink KG, Brady K, Lamb D, Collins D, Rockel TD, et al. Uptake, efficacy, and systemic distribution of naked, inhaled short interfering RNA (siRNA) and locked nucleic acid (LNA) antisense. Mol Ther 2011;19:2163-2168.
    • (2011) Mol Ther , vol.19 , pp. 2163-2168
    • Moschos, S.A.1    Frick, M.2    Taylor, B.3    Turnpenny, P.4    Graves, H.5    Spink, K.G.6    Brady, K.7    Lamb, D.8    Collins, D.9    Rockel, T.D.10
  • 36
    • 79954580272 scopus 로고    scopus 로고
    • Current prospects for RNA interference-based therapies
    • Davidson BL, McCray PB Jr. Current prospects for RNA interference-based therapies. Nat Rev Genet 2011;12:329-340.
    • (2011) Nat Rev Genet , vol.12 , pp. 329-340
    • Davidson, B.L.1    McCray Jr., P.B.2
  • 38
    • 84887259394 scopus 로고    scopus 로고
    • ClinicalTrials.gov results database. ClinicalTrials.gov Identifier: NCT01225211. National Library of Medicine (NLM) at the National Institutes of Health (NIH); [updated 2014 Feb 27]. Available from
    • ClinicalTrials.gov results database. Study of VX-809 alone and in combination with VX-770 in cystic fibrosis (CF) patients homozygous or heterozygous for the F508del-CFTR mutation. ClinicalTrials.gov Identifier: NCT01225211. National Library of Medicine (NLM) at the National Institutes of Health (NIH); 2010 [updated 2014 Feb 27]. Available from: http://www. clinicaltrials.gov/
    • (2010) Study of VX-809 Alone and in Combination with VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
  • 39
    • 1442331948 scopus 로고    scopus 로고
    • Gene expression profile analysis of 4-phenylbutyrate treatment of IB3-1 bronchial epithelial cell line demonstrates a major influence on heat-shock proteins
    • DOI 10.1152/physiolgenomics.00160.2003
    • Wright JM, Zeitlin PL, Cebotaru L, Guggino SE, Guggino WB. Gene expression profile analysis of 4-phenylbutyrate treatment of IB3-1 bronchial epithelial cell line demonstrates a major influence on heatshock proteins. Physiol Genomics 2004;16:204-211. (Pubitemid 38420037)
    • (2004) Physiological Genomics , vol.16 , pp. 204-211
    • Wright, J.M.1    Zeitlin, P.L.2    Cebotaru, L.3    Guggino, S.E.4    Guggino, W.B.5
  • 40
    • 34548154971 scopus 로고    scopus 로고
    • Additive effect of multiple pharmacological chaperones on maturation of CFTR processing mutants
    • DOI 10.1042/BJ20070478
    • Wang Y, Loo TW, Bartlett MC, Clarke DM. Additive effect of multiple pharmacological chaperones on maturation of CFTR processing mutants. Biochem J 2007;406:257-263. (Pubitemid 47310956)
    • (2007) Biochemical Journal , vol.406 , Issue.2 , pp. 257-263
    • Wang, Y.1    Loo, T.W.2    Bartlett, M.C.3    Clarke, D.M.4
  • 41
    • 84873436500 scopus 로고    scopus 로고
    • Correctors of DeltaF508 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. Correctors of DeltaF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein. FASEB J 2013;27:536-545.
    • (2013) 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
  • 43
    • 67349121012 scopus 로고    scopus 로고
    • Meta-basic estimates the size of druggable human genome
    • Plewczynski D, Rychlewski L. Meta-basic estimates the size of druggable human genome. J Mol Model 2009;15:695-699.
    • (2009) J Mol Model , vol.15 , pp. 695-699
    • Plewczynski, D.1    Rychlewski, L.2
  • 44
    • 82255164258 scopus 로고    scopus 로고
    • ProfileChaser: Searching microarray repositories based on genome-wide patterns of differential expression
    • Engreitz JM, Chen R, Morgan AA, Dudley JT, Mallelwar R, Butte AJ. ProfileChaser: searching microarray repositories based on genome-wide patterns of differential expression. Bioinformatics 2011;27:3317-3318.
    • (2011) Bioinformatics , vol.27 , pp. 3317-3318
    • Engreitz, J.M.1    Chen, R.2    Morgan, A.A.3    Dudley, J.T.4    Mallelwar, R.5    Butte, A.J.6
  • 45
    • 62349088493 scopus 로고    scopus 로고
    • GeneChaser: Identifying all biological and clinical conditions in which genes of interest are differentially expressed
    • Chen R, Mallelwar R, Thosar A, Venkatasubrahmanyam S, Butte AJ. GeneChaser: identifying all biological and clinical conditions in which genes of interest are differentially expressed. BMC Bioinformatics 2008;9:548.
    • (2008) BMC Bioinformatics , vol.9 , pp. 548
    • Chen, R.1    Mallelwar, R.2    Thosar, A.3    Venkatasubrahmanyam, S.4    Butte, A.J.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.