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Volumn 372, Issue 9640, 2008, Pages 691-692

Ignoring the nonsense: a phase II trial in cystic fibrosis

Author keywords

[No Author keywords available]

Indexed keywords

AMINOGLYCOSIDE; CYCLIC AMP; EPITHELIAL SODIUM CHANNEL; GENTAMICIN; PLACEBO; PTC 124; TRANSMEMBRANE CONDUCTANCE REGULATOR;

EID: 50149091319     PISSN: 01406736     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0140-6736(08)61169-1     Document Type: Note
Times cited : (9)

References (13)
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  • 3
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    • Evidence for airway surface dehydration as the initiating event in CF airway disease
    • Boucher R.C. Evidence for airway surface dehydration as the initiating event in CF airway disease. J Intern Med 261 (2007) 5-16
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    • Boucher, R.C.1
  • 4
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    • Population variation of common cystic fibrosis mutations
    • The Cystic Fibrosis Genetic Analysis Consortium
    • The Cystic Fibrosis Genetic Analysis Consortium. Population variation of common cystic fibrosis mutations. Hum Mutat 4 (1994) 167-177
    • (1994) Hum Mutat , vol.4 , pp. 167-177
  • 5
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    • Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis
    • Johnson L.G., Olsen J.C., Sarkadi B., Moore K.L., Swanstrom R., and Boucher R.C. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 2 (1992) 21-25
    • (1992) Nat Genet , vol.2 , pp. 21-25
    • Johnson, L.G.1    Olsen, J.C.2    Sarkadi, B.3    Moore, K.L.4    Swanstrom, R.5    Boucher, R.C.6
  • 6
    • 0029994529 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations
    • Howard M., Frizzell R.A., and Bedwell D.M. Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations. Nat Med 2 (1996) 467-469
    • (1996) Nat Med , vol.2 , pp. 467-469
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  • 7
    • 0034961464 scopus 로고    scopus 로고
    • Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis
    • Clancy J.P., 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 163 (2001) 1683-1692
    • (2001) Am J Respir Crit Care Med , vol.163 , pp. 1683-1692
    • Clancy, J.P.1    Bebok, Z.2    Ruiz, F.3
  • 8
    • 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 349 (2003) 1433-1441
    • (2003) N Engl J Med , vol.349 , pp. 1433-1441
    • Wilschanski, M.1    Yahav, Y.2    Yaacov, Y.3
  • 10
    • 0033858002 scopus 로고    scopus 로고
    • A biochemical basis for the inherited susceptibility to aminoglycoside ototoxicity
    • Guan M.X., Fischel-Ghodsian N., and Attardi G. A biochemical basis for the inherited susceptibility to aminoglycoside ototoxicity. Hum Mol Genet 9 (2000) 1787-1793
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  • 11
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    • PTC124 targets genetic disorders caused by nonsense mutations
    • Welch E.M., Barton E.R., Zhuo J., et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 447 (2007) 87-91
    • (2007) Nature , vol.447 , pp. 87-91
    • Welch, E.M.1    Barton, E.R.2    Zhuo, J.3
  • 12
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    • Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers
    • Hirawat S., Welch E.M., Elfring G.L., et al. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol 47 (2007) 430-444
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  • 13
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    • Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial
    • 10.1016/S0140-6736(08)61168-X published online Aug 21.
    • 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) 10.1016/S0140-6736(08)61168-X published online Aug 21.
    • (2008) Lancet
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* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.