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注意：仅列出部分参考文献，下载原文获取全部文献信息。CFTR modulator theratyping: Current status, gaps and future directions
John Paul Clancy et al.
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VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
J. C. Davies et al.
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Nikhil T. Awatade et al.
FRONTIERS IN PHARMACOLOGY (2018)
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Chi B. Vu et al.
JOURNAL OF MEDICINAL CHEMISTRY (2017)
Effect of bronchodilators in healthy individuals receiving lumacaftor/ivaca ftor combination therapy
Gautham Marigowda et al.
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Real-life acute lung function changes after lumacaftor/ivacaftor first administration in pediatric patients with cystic fibrosis
Aurelie Labaste et al.
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Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease
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A common mechanism for CFTR potentiators
Han-I Yeh et al.
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Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis
Steven M. Rowe et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del
Jennifer L. Taylor-Cousar et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled, phase 3 trial
Felix Ratjen et al.
LANCET RESPIRATORY MEDICINE (2017)
An Observational Study of Outcomes and Tolerances in Patients with Cystic Fibrosis Initiated on Lumacaftor/Ivacaftor
Mark T. Jennings et al.
ANNALS OF THE AMERICAN THORACIC SOCIETY (2017)
Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor)
Elizabeth Matthes et al.
BRITISH JOURNAL OF PHARMACOLOGY (2016)
Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis
J. Stuart Elborn et al.
LANCET RESPIRATORY MEDICINE (2016)
Synergistic Potentiation of Cystic Fibrosis Transmembrane Conductance Regulator Gating by Two Chemically Distinct Potentiators, Ivacaftor (VX-770) and 5-Nitro-2-(3-Phenylpropylamino) Benzoate
Wen-Ying Lin et al.
MOLECULAR PHARMACOLOGY (2016)
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
C. E. Wainwright et al.
NEW ENGLAND JOURNAL OF MEDICINE (2015)
Cystic fibrosis genetics: from molecular understanding to clinical application
Garry R. Cutting
NATURE REVIEWS GENETICS (2015)
Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels
Z. Kopeikin et al.
JOURNAL OF CYSTIC FIBROSIS (2014)
Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression
Guido Veit et al.
SCIENCE TRANSLATIONAL MEDICINE (2014)
Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis
Deborah M. Cholon et al.
SCIENCE TRANSLATIONAL MEDICINE (2014)
VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
Hong Yu Ren et al.
MOLECULAR BIOLOGY OF THE CELL (2013)
Mechanism-based corrector combination restores ΔF508-CFTR folding and function
Tsukasa Okiyoneda et al.
NATURE CHEMICAL BIOLOGY (2013)
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
J. P. Clancy et al.
THORAX (2012)
CFTR: folding, misfolding and correcting the ΔF508 conformational defect
Gergely L. Lukacs et al.
TRENDS IN MOLECULAR MEDICINE (2012)
Targeting F508del-CFTR to develop rational new therapies for cystic fibrosis
Zhi-wei Cai et al.
ACTA PHARMACOLOGICA SINICA (2011)
Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
Fredrick Van Goor et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)
Peripheral Protein Quality Control Removes Unfolded CFTR from the Plasma Membrane
Tsukasa Okiyoneda et al.
SCIENCE (2010)
Gating of the CFTR Cl- channel by ATP-driven nucleotide-binding domain dimerisation
Tzyh-Chang Hwang et al.
JOURNAL OF PHYSIOLOGY-LONDON (2009)
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
Fredrick Van Goor et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Sequential quality-control checkpoints triage misfolded cystic fibrosis transmembrane conductance regulator
J. Michael Younger et al.
CELL (2006)
Towards an in vitro model of cystic fibrosis small airway epithelium: characterisation of the human bronchial epithelial cell line CFBE41o-
C Ehrhardt et al.
CELL AND TISSUE RESEARCH (2006)
The F508 cystic fibrosis mutation impairs domain-domain interactions and arrests post-translational folding of CFTR
K Du et al.
NATURE STRUCTURAL & MOLECULAR BIOLOGY (2005)
Misfolding diverts CFTR from recycling to degradation: quality control at early endosomes
M Sharma et al.
JOURNAL OF CELL BIOLOGY (2004)
The Hsc70 co-chaperone CHIP targets immature CFTR for proteasomal degradation
GC Meacham et al.
NATURE CELL BIOLOGY (2001)
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