Mechanism of CFTR correction by type I folding correctors

Co-Translational Folding of the First Transmembrane Domain of ABC-Transporter CFTR is Supported by Assembly with the First Cytosolic Domain

(2021) Bertrand Kleizen et al.   JOURNAL OF MOLECULAR BIOLOGY

Discovery of a picomolar potency pharmacological corrector of the mutant CFTR chloride channel

(2020) Nicoletta Pedemonte et al.   Science Advances

Second-Generation Pharmacological Chaperones: Beyond Inhibitors

(2020) My Lan Tran et al.   MOLECULES

Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination

(2020) Guido Veit et al.   JCI Insight

Structural identification of a hotspot on CFTR for potentiation

(2019) Fangyu Liu et al.   SCIENCE

A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis

(2019) Al-Rahim R. Habib et al.   Scientific Reports

CFTR transmembrane segments are impaired in their conformational adaptability by a pathogenic loop mutation and dynamically stabilized by Lumacaftor

(2019) Georg Krainer et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

(2018) Xueqing Wang et al.   JOURNAL OF MEDICINAL CHEMISTRY

Molecular Mechanism of Action of Trimethylangelicin Derivatives as CFTR Modulators

(2018) Onofrio Laselva et al.   Frontiers in Pharmacology

Structure-guided combination therapy to potently improve the function of mutant CFTRs

(2018) Guido Veit et al.   NATURE MEDICINE

Structure, Gating, and Regulation of the CFTR Anion Channel

(2018) László Csanády et al.   PHYSIOLOGICAL REVIEWS

Molecular structure of the ATP-bound, phosphorylated human CFTR

(2018) Zhe Zhang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation

(2017) Xin Meng et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

(2017) Rhea P. Hudson et al.   MOLECULAR PHARMACOLOGY

MotionCor2: anisotropic correction of beam-induced motion for improved cryo-electron microscopy

(2017) Shawn Q Zheng et al.   NATURE METHODS

Atomic Structure of the Cystic Fibrosis Transmembrane Conductance Regulator

(2016) Zhe Zhang et al.   CELL

From the endoplasmic reticulum to the plasma membrane: mechanisms of CFTR folding and trafficking

(2016) Carlos M. Farinha et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Gctf: Real-time CTF determination and correction

(2016) Kai Zhang   JOURNAL OF STRUCTURAL BIOLOGY

Capturing the Direct Binding of CFTR Correctors to CFTR by Using Click Chemistry

(2015) Chandrima Sinha et al.   CHEMBIOCHEM

CTFFIND4: Fast and accurate defocus estimation from electron micrographs

(2015) Alexis Rohou et al.   JOURNAL OF STRUCTURAL BIOLOGY

VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface

(2014) Paul D.W. Eckford et al.   CHEMISTRY & BIOLOGY

Direct interaction of a CFTR potentiator and a CFTR corrector with phospholipid bilayers

(2014) Debora Baroni et al.   EUROPEAN BIOPHYSICS JOURNAL WITH BIOPHYSICS LETTERS

Screening and large-scale expression of membrane proteins in mammalian cells for structural studies

(2014) April Goehring et al.   Nature Protocols

Cystic fibrosis genetics: from molecular understanding to clinical application

(2014) Garry R. Cutting   NATURE REVIEWS GENETICS

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation

(2013) Jane C. Davies et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Corrector VX-809 stabilizes the first transmembrane domain of CFTR

(2013) Tip W. Loo et al.   BIOCHEMICAL PHARMACOLOGY

Revertants, Low Temperature, and Correctors Reveal the Mechanism of F508del-CFTR Rescue by VX-809 and Suggest Multiple Agents for Full Correction

(2013) Carlos M. Farinha et al.   CHEMISTRY & BIOLOGY

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1

(2013) Hong Yu Ren et al.   MOLECULAR BIOLOGY OF THE CELL

Mechanism-based corrector combination restores ΔF508-CFTR folding and function

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

Vx-770 potentiates CFTR function by promoting decoupling between the gating cycle and ATP hydrolysis cycle

(2013) Kang-Yang Jih et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

HGF Stimulation of Rac1 Signaling Enhances Pharmacological Correction of the Most Prevalent Cystic Fibrosis Mutant F508del-CFTR

(2012) Sónia Moniz et al.   ACS Chemical Biology

Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein

(2012) Lihua He et al.   FASEB JOURNAL

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

(2012) Paul D. W. Eckford et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

RELION: Implementation of a Bayesian approach to cryo-EM structure determination

(2012) Sjors H.W. Scheres   JOURNAL OF STRUCTURAL BIOLOGY

Fiji: an open-source platform for biological-image analysis

(2012) Johannes Schindelin et al.   NATURE METHODS

Alteration of CFTR transmembrane span integration by disease-causing mutations

(2011) Anna E. Patrick et al.   MOLECULAR BIOLOGY OF THE CELL

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

(2011) Gergely L. Lukacs et al.   TRENDS IN MOLECULAR MEDICINE

PHENIX: a comprehensive Python-based system for macromolecular structure solution

(2010) Paul D. Adams et al.   ACTA CRYSTALLOGRAPHICA SECTION D-BIOLOGICAL CRYSTALLOGRAPHY

Lipophilicity and Its Relationship with Passive Drug Permeation

(2010) Xiangli Liu et al.   PHARMACEUTICAL RESEARCH

electronic Ligand Builder and Optimization Workbench(eLBOW): a tool for ligand coordinate and restraint generation

(2009) Nigel W. Moriarty et al.   ACTA CRYSTALLOGRAPHICA SECTION D-BIOLOGICAL CRYSTALLOGRAPHY

MolProbity: all-atom structure validation for macromolecular crystallography

(2009) Vincent B. Chen et al.   ACTA CRYSTALLOGRAPHICA SECTION D-BIOLOGICAL CRYSTALLOGRAPHY

Simple and efficient site-directed mutagenesis using two single-primer reactions in parallel to generate mutants for protein structure-function studies

(2009) Oded Edelheit et al.   BMC BIOTECHNOLOGY

Similar Energetic Contributions of Packing in the Core of Membrane and Water-Soluble Proteins

(2009) Nathan H. Joh et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Cooperative Assembly and Misfolding of CFTR Domains In Vivo

(2009) Kai Du et al.   MOLECULAR BIOLOGY OF THE CELL

SPIDER image processing for single-particle reconstruction of biological macromolecules from electron micrographs

(2009) Tanvir R Shaikh et al.   Nature Protocols

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Assembly and Misassembly of Cystic Fibrosis Transmembrane Conductance Regulator: Folding Defects Caused by Deletion of F508 Occur Before and After the Calnexin-dependent Association of Membrane Spanning Domain (MSD) 1 and MSD2

(2008) Meredith F. N. Rosser et al.   MOLECULAR BIOLOGY OF THE CELL

Phenylalanine-508 mediates a cytoplasmic-membrane domain contact in the CFTR 3D structure crucial to assembly and channel function

(2008) A. W. R. Serohijos et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA