Chronic ivacaftor treatment: Getting F508del-CFTR into more trouble?

被引:6
作者
Mall, Marcus A. [1 ,2 ,3 ]
Sheppard, David N. [4 ]
机构
[1] Heidelberg Univ, Translat Lung Res Ctr Heidelberg TLRC, Dept Translat Pulmonol, D-69120 Heidelberg, Germany
[2] Heidelberg Univ, Div Pediat Pulmonol, D-69120 Heidelberg, Germany
[3] Heidelberg Univ, Cyst Fibrosis Ctr, D-69120 Heidelberg, Germany
[4] Univ Bristol, Sch Physiol & Pharmacol, Bristol BS8 1TD, Avon, England
关键词
CYSTIC-FIBROSIS; CFTR POTENTIATOR; IN-VITRO; MUTATION; THERAPY; MODELS;
D O I
10.1016/j.jcf.2014.10.001
中图分类号
R56 [呼吸系及胸部疾病];
学科分类号
摘要
引用
收藏
页码:605 / 607
页数:3
相关论文
共 16 条
  • [1] Pharmacological therapy for cystic fibrosis: From bench to bedside
    Becq, Frederic
    Mall, Marcus A.
    Sheppard, David N.
    Conese, Massimo
    Zegarra-Moran, Olga
    [J]. JOURNAL OF CYSTIC FIBROSIS, 2011, 10 : S129 - S145
  • [2] CFTR functional measurements in human models for diagnosis, prognosis and personalized therapy: Report on the pre-conference meeting to the 11th ECFS Basic Science Conference, Malta, 26-29 March 2014
    Beekman, Jeffrey M.
    Sermet-Gaudelus, Isabelle
    de Boeck, Kris
    Gonska, Tanja
    Derichs, Nico
    Mall, Marcus A.
    Mehta, Anil
    Martin, Ulrich
    Drumm, Mitch
    Amaral, Margarida D.
    [J]. JOURNAL OF CYSTIC FIBROSIS, 2014, 13 (04) : 363 - 372
  • [3] 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, Michael P.
    Bell, Scott C.
    Konstan, Michael W.
    McColley, Susanna A.
    Rowe, Steven M.
    Rietschel, Ernst
    Huang, Xiaohong
    Waltz, David
    Patel, Naimish R.
    Rodman, David
    [J]. LANCET RESPIRATORY MEDICINE, 2014, 2 (07) : 527 - 538
  • [4] Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis
    Cholon, Deborah M.
    Quinney, Nancy L.
    Fulcher, M. Leslie
    Esther, Charles R., Jr.
    Das, Jhuma
    Dokholyan, Nikolay V.
    Randell, Scott H.
    Boucher, Richard C.
    Gentzsch, Martina
    [J]. SCIENCE TRANSLATIONAL MEDICINE, 2014, 6 (246)
  • [5] Sweat gland bioelectrics differ in cystic fibrosis: a new concept for potential diagnosis and assessment of CFTR function in cystic fibrosis
    Gonska, T.
    Ip, W.
    Turner, D.
    Han, W. S.
    Rose, J.
    Durie, P.
    Quinton, P.
    [J]. THORAX, 2009, 64 (11) : 932 - 938
  • [6] CFTR Cl- channel function in native human colon correlates with the genotype and phenotype in cystic fibrosis
    Hirtz, S
    Gonska, T
    Seydewitz, HH
    Thomas, J
    Greiner, P
    Kuehr, J
    Brandis, M
    Eichler, I
    Rocha, H
    Lopes, AI
    Barreto, C
    Ramalho, A
    Amaral, MD
    Kunzelmann, K
    Mall, M
    [J]. GASTROENTEROLOGY, 2004, 127 (04) : 1085 - 1095
  • [7] CFTR: cystic fibrosis and beyond
    Mall, Marcus A.
    Hartl, Dominik
    [J]. EUROPEAN RESPIRATORY JOURNAL, 2014, 44 (04) : 1042 - 1054
  • [8] A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
    Ramsey, Bonnie W.
    Davies, Jane
    McElvaney, N. Gerard
    Tullis, Elizabeth
    Bell, Scott C.
    Drevinek, Pavel
    Griese, Matthias
    McKone, Edward F.
    Wainwright, Claire E.
    Konstan, Michael W.
    Moss, Richard
    Ratjen, Felix
    Sermet-Gaudelus, Isabelle
    Rowe, Steven M.
    Dong, Qunming
    Rodriguez, Sally
    Yen, Karl
    Ordonez, Claudia
    Elborn, J. Stuart
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2011, 365 (18) : 1663 - 1672
  • [9] The K+ Channel Opener 1-EBIO Potentiates Residual Function of Mutant CFTR in Rectal Biopsies from Cystic Fibrosis Patients
    Roth, Eva K.
    Hirtz, Stephanie
    Duerr, Julia
    Wenning, Daniel
    Eichler, Irmgard
    Seydewitz, Hans H.
    Amaral, Margarida D.
    Mall, Marcus A.
    [J]. PLOS ONE, 2011, 6 (08):
  • [10] Prolonged treatment of cells with genistein modulates the expression and function of the cystic fibrosis transmembrane conductance regulator
    Schmidt, A.
    Hughes, L. K.
    Cai, Z.
    Mendes, F.
    Li, H.
    Sheppard, D. N.
    Amaral, M. D.
    [J]. BRITISH JOURNAL OF PHARMACOLOGY, 2008, 153 (06) : 1311 - 1323