相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN)
Silke van Koningsbruggen-Rietschel et al.
JOURNAL OF CYSTIC FIBROSIS (2020)
Biological Characterization of F508delCFTR Protein Processing by the CFTR Corrector ABBV-2222/GLPG2222
Ashvani K. Singh et al.
JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2020)
Amplifiers co-translationally enhance CFTR biosynthesis via PCBP1-mediated regulation of CFTR mRNA
Danijela Dukovski et al.
JOURNAL OF CYSTIC FIBROSIS (2020)
Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis
Gitte Berkers et al.
CELL REPORTS (2019)
GLPG1837, a CFTR potentiator, in p.Gly551Asp (G551D)-CF patients: An open-label, single-arm, phase 2a study (SAPHIRA1)
Jane C. Davies et al.
JOURNAL OF CYSTIC FIBROSIS (2019)
CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials
Scott C. Bell et al.
JOURNAL OF CYSTIC FIBROSIS (2019)
Identification of GLPG/ABBV-2737, a Novel Class of Corrector, Which Exerts Functional Synergy With Other CFTR Modulators
Gert de Wilde et al.
FRONTIERS IN PHARMACOLOGY (2019)
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis
Seth Walker et al.
JOURNAL OF CYSTIC FIBROSIS (2019)
Identifying the molecular target sites for CFTR potentiators GLPG1837 and VX-770
Han- Yeh et al.
JOURNAL OF GENERAL PHYSIOLOGY (2019)
Structural identification of a hotspot on CFTR for potentiation
Fangyu Liu et al.
SCIENCE (2019)
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial
Harry G. M. Heijerman et al.
LANCET (2019)
Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
P. G. Middleton et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
Discovery of ABBV/GLPG-3221, a Potent Corrector of CFTR for the Treatment of Cystic Fibrosis
Marc J. C. Scanio et al.
ACS MEDICINAL CHEMISTRY LETTERS (2019)
Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response?
Iwona Pranke et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2019)
Predictive factors for lumacaftor/ivacaftor clinical response
Alexandra Masson et al.
JOURNAL OF CYSTIC FIBROSIS (2019)
Use of a High-Throughput Phenotypic Screening Strategy to Identify Amplifiers, a Novel Pharmacological Class of Small Molecules That Exhibit Functional Synergy with Potentiators and Correctors
Kenneth A. Giuliano et al.
SLAS DISCOVERY (2018)
Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR
Scott H. Donaldson et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2018)
Discovery of 4-[(2R,4R)-4-({[1-(2,2-Difluoro-1,3-benzodioxol-5-yl)cyclopropyl]carbonyl}amino)-7-(difluoromethoxy)-3,4-dihydro-2H-chromen-2-yl]benzoic Acid (ABBV/GLPG-2222), a Potent Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Corrector for the Treatment of Cystic Fibrosis
Xueqing Wang et al.
JOURNAL OF MEDICINAL CHEMISTRY (2018)
Discovery of N-(3-Carbamoyl-5,5,7,7-tetramethyl-5,7-dihydro-4H-thieno[2,3-c]pyran-2-yl)-lH-pyrazole-5-carboxamide (GLPG1837), a Novel Potentiator Which Can Open Class III Mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Channels to a High Extent
Steven E. Van der Plas et al.
JOURNAL OF MEDICINAL CHEMISTRY (2018)
Correctors of the Major Cystic Fibrosis Mutant Interact through Membrane-Spanning Domains
Onofrio Laselva et al.
MOLECULAR PHARMACOLOGY (2018)
Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study
Margaret Rosenfeld et al.
LANCET RESPIRATORY MEDICINE (2018)
VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
Dominic Keating et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
J. C. Davies et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Identification and Characterization of Novel CFTR Potentiators
Maarten Gees et al.
FRONTIERS IN PHARMACOLOGY (2018)
Functional Defect of Variants in the Adenosine Triphosphate-Binding Sites of ABCB4 and Their Rescue by the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator, Ivacaftor ( VX-770)
Jean-Louis Delaunay et al.
HEPATOLOGY (2017)
Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease
Dominique Hubert et al.
JOURNAL OF CYSTIC FIBROSIS (2017)
Altering Metabolic Profiles of Drugs by Precision Deuteration 2: Discovery of a Deuterated Analog of Ivacaftor with Differentiated Pharmacokinetics for Clinical Development
Scott L. Harbeson et al.
JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2017)
Direct Binding of the Corrector VX-809 to Human CFTR NBD1: Evidence of an Allosteric Coupling between the Binding Site and the NBD1:CL4 Interface
Rhea P. Hudson et al.
MOLECULAR PHARMACOLOGY (2017)
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)
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators
Iwona M. Pranke et al.
SCIENTIFIC REPORTS (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)
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study
Michael W. Konstan 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)
Orkambi® and amplifier co-therapy improves function from a rare CFTR mutation in gene-edited cells and patient tissue
Steven V. Molinski et al.
EMBO MOLECULAR MEDICINE (2017)
Lumacaftor/Ivacaftor in Patients Aged 6-11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR
Carlos E. Milla et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2017)
Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR
Steven M. Rowe et al.
ANNALS OF THE AMERICAN THORACIC SOCIETY (2017)
Leveraging Rules of Nonsense-Mediated mRNA Decay for Genome Engineering and Personalized Medicine
Maximilian W. Popp et al.
CELL (2016)
Cystic fibrosis: a model system for precision medicine
Stacey L. Martiniano et al.
CURRENT OPINION IN PEDIATRICS (2016)
Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States
Jennifer Taylor-Cousar et al.
JOURNAL OF CYSTIC FIBROSIS (2016)
From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations
Gudio Veit et al.
MOLECULAR BIOLOGY OF THE CELL (2016)
Therapeutic challenges posed by critical drug-drug interactions in cystic fibrosis
Cameron L. Jordan et al.
PEDIATRIC PULMONOLOGY (2016)
Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study
Jane C. Davies et al.
LANCET RESPIRATORY MEDICINE (2016)
Inflammation in cystic fibrosis lung disease: Pathogenesis and therapy
Andre M. Cantin et al.
JOURNAL OF CYSTIC FIBROSIS (2015)
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
C. E. Wainwright et al.
NEW ENGLAND JOURNAL OF MEDICINE (2015)
Biosynthesis of cystic fibrosis transmembrane conductance regulator
Iwona M. Pranke et al.
INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY (2014)
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation
Kris De Boeck et al.
JOURNAL OF CYSTIC FIBROSIS (2014)
Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis
Deborah M. Cholon et al.
SCIENCE TRANSLATIONAL MEDICINE (2014)
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
Michael P. Boyle et al.
LANCET RESPIRATORY MEDICINE (2014)
Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation
Jane C. Davies et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2013)
Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
Patrick A. Flume et al.
CHEST (2012)
Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a Phosphorylation-dependent but ATP-independent Manner
Paul D. W. Eckford et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2012)
Reduced airway surface pH impairs bacterial killing in the porcine cystic fibrosis lung
Alejandro A. Pezzulo et al.
NATURE (2012)
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)
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)
Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis
Peter A. Sloane et al.
CURRENT OPINION IN PULMONARY MEDICINE (2010)
Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation
Frank J. Accurso et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Gating of the CFTR Cl- channel by ATP-driven nucleotide-binding domain dimerisation
Tzyh-Chang Hwang et al.
JOURNAL OF PHYSIOLOGY-LONDON (2009)
ATP hydrolysis-driven gating in cystic fibrosis transmembrane conductance regulator
Daniella Muallem et al.
PHILOSOPHICAL TRANSACTIONS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES (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)
CFTR function and prospects for therapy
John R. Riordan
ANNUAL REVIEW OF BIOCHEMISTRY (2008)
CFTR channel opening by ATP-driven tight dimerization of its nucleotide-binding domains
P Vergani et al.
NATURE (2005)
Misfolding diverts CFTR from recycling to degradation: quality control at early endosomes
M Sharma et al.
JOURNAL OF CELL BIOLOGY (2004)
Nonsense-mediated mRNA decay: Splicing, translation and mRNP dynamics
LE Maquat
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2004)
Efficient intracellular processing of the endogenous cystic fibrosis transmembrane conductance regulator in epithelial cell lines
K Varga et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2004)
A mutation in the cystic fibrosis transmembrane conductance regulator generates a novel internalization sequence and enhances endocytic rates
MR Silvis et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2003)
A principal role for the proteasome in endoplasmic reticulum-associated degradation of misfolded intracellular cystic fibrosis transmembrane conductance regulator
MS Gelman et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2002)
分享论文
