Related references
Note: Only part of the references are listed.Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More F508del Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial
Matthias Griese et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2021)
Modulators of CFTR. Updates on clinical development and future directions
Emmanuelle Bardin et al.
EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY (2021)
Rescue of multiple class II CFTR mutations by elexacaftor plus tezacaftor plus ivacaftor mediated in part by the dual activities of elexacaftor as both corrector and potentiator
Onofrio Laselva et al.
EUROPEAN RESPIRATORY JOURNAL (2021)
Effect of CFTR correctors on the traffic and the function of intracellularly retained ABCB4 variants
Amel Ben Saad et al.
LIVER INTERNATIONAL (2021)
Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery
Marcello Carotti et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)
Discovery of a picomolar potency pharmacological corrector of the mutant CFTR chloride channel
Nicoletta Pedemonte et al.
SCIENCE ADVANCES (2020)
CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine
Miqueias Lopes-Pacheco
FRONTIERS IN PHARMACOLOGY (2020)
Current development of CFTR potentiators in the last decade
Virginia Spano et al.
EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY (2020)
Distinctive lipid signatures of bronchial epithelial cells associated with cystic fibrosis drugs, including Trikafta
Nara Liessi et al.
JCI INSIGHT (2020)
From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis
Andrea Gramegna et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)
Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination
Guido Veit et al.
JCI INSIGHT (2020)
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)
STRUCTURE, GATING, AND REGULATION OF THE CFTR ANION CHANNEL
Laszlo Csanady et al.
PHYSIOLOGICAL REVIEWS (2019)
High-throughput screening identifies FAU protein as a regulator of mutant cystic fibrosis transmembrane conductance regulator channel
Valeria Tomati et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2018)
Therapeutic approaches to CFTR dysfunction: From discovery to drug development
Hongyu Li et al.
JOURNAL OF CYSTIC FIBROSIS (2018)
The structural basis of cystic fibrosis
Xin Meng et al.
BIOCHEMICAL SOCIETY TRANSACTIONS (2018)
Structure-guided combination therapy to potently improve the function of mutant CFTRs
Guido Veit et al.
NATURE 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)
Pharmacological Inhibition of the Ubiquitin Ligase RNF5 Rescues F508del-CFTR in Cystic Fibrosis Airway Epithelia
Elvira Sondo et al.
CELL CHEMICAL BIOLOGY (2018)
Thymosin alpha-1 does not correct F508del-CFTR in cystic fibrosis airway epithelia
Valeria Tomati et al.
JCI INSIGHT (2018)
Cystic fibrosis: a clinical view
Carlo Castellani et al.
CELLULAR AND MOLECULAR LIFE SCIENCES (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 Patients with Cystic Fibrosis Homozygous for Phe508del
Jennifer L. Taylor-Cousar et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Corrector VX-809 promotes interactions between cytoplasmic loop one and the first nucleotide-binding domain of CFTR
Tip W. Loo et al.
BIOCHEMICAL PHARMACOLOGY (2017)
Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression Defect
Guido Veit et al.
PLOS BIOLOGY (2016)
Rescuing Trafficking Mutants of the ATP-binding Cassette Protein, ABCA4, with Small Molecule Correctors as a Treatment for Stargardt Eye Disease
Inna Sabirzhanova et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2015)
Corrector VX-809 stabilizes the first transmembrane domain of CFTR
Tip W. Loo et al.
BIOCHEMICAL PHARMACOLOGY (2013)
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)
Association of TMEM16A chloride channel overexpression with airway goblet cell metaplasia
Paolo Scudieri et al.
JOURNAL OF PHYSIOLOGY-LONDON (2012)
Rescue of the mutant CFTR chloride channel by pharmacological correctors and low temperature analyzed by gene expression profiling
Elvira Sondo et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2011)
Dual Activity of Aminoarylthiazoles on the Trafficking and Gating Defects of the Cystic Fibrosis Transmembrane Conductance Regulator Chloride Channel Caused by Cystic Fibrosis Mutations
Nicoletta Pedemonte et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (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)
Influence of cell background on pharmacological rescue of mutant CFTR
Nicoletta Pedemonte et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2010)
Enhanced cell-surface stability of rescued ΔF508 cystic fibrosis transmembrane conductance regulator (CFTR) by pharmacological chaperones
Karoly Varga et al.
BIOCHEMICAL JOURNAL (2008)
Small-molecule correctors of defective ΔF508-CFTR cellular processing identified by high-throughput screening
N Pedemonte et al.
JOURNAL OF CLINICAL INVESTIGATION (2005)
Conformational and temperature-sensitive stability defects of the ΔF508 cystic fibrosis transmembrane conductance regulator in post-endoplasmic reticulum compartments
M Sharma et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2001)
The Delta F508 mutation shortens the biochemical half-life of plasma membrane CFTR in polarized epithelial cells
GD Heda et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2001)
Share Paper
