CFTR pharmacology

Cystic Fibrosis Transmembrane Conductance Regulator in Sarcoplasmic Reticulum of Airway Smooth Muscle. Implications for Airway Contractility

(2016) Daniel P. Cook et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Cystic fibrosis

(2016) J Stuart Elborn   LANCET

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

(2016) Gudio Veit et al.   MOLECULAR BIOLOGY OF THE CELL

Synthesis and structure–activity relationship of aminoarylthiazole derivatives as correctors of the chloride transport defect in cystic fibrosis

(2015) Emanuela Pesce et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

∆F508 CFTR interactome remodelling promotes rescue of cystic fibrosis

(2015) Sandra Pankow et al.   NATURE

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

Unravelling druggable signalling networks that control F508del-CFTR proteostasis

(2015) Ramanath Narayana Hegde et al.   eLife

Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation

(2014) Daniela De Stefano et al.   Autophagy

Roscovitine is a proteostasis regulator that corrects the trafficking defect of F508del-CFTR by a CDK-independent mechanism

(2014) C Norez et al.   BRITISH JOURNAL OF PHARMACOLOGY

Full-open and closed CFTR channels, with lateral tunnels from the cytoplasm and an alternative position of the F508 region, as revealed by molecular dynamics

(2014) Jean-Paul Mornon et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Discovery of N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, Ivacaftor), a Potent and Orally Bioavailable CFTR Potentiator

(2014) Sabine Hadida et al.   JOURNAL OF MEDICINAL CHEMISTRY

Constrained Bithiazoles: Small Molecule Correctors of Defective ΔF508–CFTR Protein Trafficking

(2014) Keith C. Coffman et al.   JOURNAL OF MEDICINAL CHEMISTRY

Some gating potentiators, including VX-770, diminish  F508-CFTR functional expression

(2014) G. Veit et al.   Science Translational Medicine

Potentiator ivacaftor abrogates pharmacological correction of  F508 CFTR in cystic fibrosis

(2014) D. M. Cholon et al.   Science Translational Medicine

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

Acquired Cystic Fibrosis Transmembrane Conductance Regulator Dysfunction in the Lower Airways in COPD

(2013) Mark T. Dransfield et al.   CHEST

Chloride channel-targeted therapy for secretory diarrheas

(2013) Jay R Thiagarajah et al.   CURRENT OPINION IN PHARMACOLOGY

Discovery of novel potent ΔF508-CFTR correctors that target the nucleotide binding domain

(2013) Norbert Odolczyk et al.   EMBO Molecular Medicine

Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function

(2013) Fredrick Van Goor et al.   Journal of Cystic Fibrosis

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

Pseudohalide anions reveal a novel extracellular site for potentiators to increase CFTR function

(2012) Man-Song Li et al.   BRITISH JOURNAL OF PHARMACOLOGY

A potentiator induces conformational changes on the recombinant CFTR nucleotide binding domains in solution

(2012) Elena Galfrè et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Correction of F508del-CFTR Trafficking by the Sponge Alkaloid Latonduine Is Modulated by Interaction with PARP

(2012) Graeme W. Carlile et al.   CHEMISTRY & BIOLOGY

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

Abnormal endocrine pancreas function at birth in cystic fibrosis ferrets

(2012) Alicia K. Olivier et al.   JOURNAL OF CLINICAL INVESTIGATION

Use of Kinase Inhibitors to Correct ΔF508-CFTR Function

(2012) Agata M. Trzcińska-Daneluti et al.   MOLECULAR & CELLULAR PROTEOMICS

A Pharmacologic Approach to Acquired Cystic Fibrosis Transmembrane Conductance Regulator Dysfunction in Smoking Related Lung Disease

(2012) Peter A. Sloane et al.   PLoS One

Dual Activity of Aminoarylthiazoles on the Trafficking and Gating Defects of the Cystic Fibrosis Transmembrane Conductance Regulator Chloride Channel Caused by Cystic Fibrosis Mutations

(2011) Nicoletta Pedemonte et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Potent, Metabolically Stable Benzopyrimido-pyrrolo-oxazine-dione (BPO) CFTR Inhibitors for Polycystic Kidney Disease

(2011) David S. Snyder et al.   JOURNAL OF MEDICINAL CHEMISTRY

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for theF508del-CFTRmutation

(2011) J P Clancy et al.   THORAX

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

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

Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis

(2010) Xingshen Sun et al.   JOURNAL OF CLINICAL INVESTIGATION

On the mechanism of CFTR inhibition by a thiazolidinone derivative

(2010) Zoia Kopeikin et al.   JOURNAL OF GENERAL PHYSIOLOGY

Cystic Fibrosis Transmembrane Regulator Inhibitors CFTRinh-172 and GlyH-101 Target Mitochondrial Functions, Independently of Chloride Channel Inhibition

(2010) M. Kelly et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Correction of the  Phe508 Cystic Fibrosis Transmembrane Conductance Regulator Trafficking Defect by the Bioavailable Compound Glafenine

(2010) R. Robert et al.   MOLECULAR PHARMACOLOGY

Defective CFTR induces aggresome formation and lung inflammation in cystic fibrosis through ROS-mediated autophagy inhibition

(2010) Alessandro Luciani et al.   NATURE CELL BIOLOGY

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

(2010) Frank J. Accurso et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cystic Fibrosis Pigs Develop Lung Disease and Exhibit Defective Bacterial Eradication at Birth

(2010) D. A. Stoltz et al.   Science Translational Medicine

Nanomolar Potency Pyrimido-pyrrolo-quinoxalinedione CFTR Inhibitor Reduces Cyst Size in a Polycystic Kidney Disease Model

(2009) Lukmanee Tradtrantip et al.   JOURNAL OF MEDICINAL CHEMISTRY

Mechanisms for Rescue of Correctable Folding Defects in CFTRΔF508

(2009) Diane E. Grove et al.   MOLECULAR BIOLOGY OF THE CELL

A Small-Molecule Modulator Interacts Directly with  Phe508-CFTR to Modify Its ATPase Activity and Conformational Stability

(2009) L. Wellhauser et al.   MOLECULAR PHARMACOLOGY

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis

(2009) Darren M Hutt et al.   Nature Chemical Biology

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

CFTR Function and Prospects for Therapy

(2008) John R. Riordan   Annual Review of Biochemistry

Evidence for direct CFTR inhibition by CFTRinh-172 based on Arg347mutagenesis

(2008) Emanuela Caci et al.   BIOCHEMICAL JOURNAL

Thiazolidinone CFTR inhibitors with improved water solubility identified by structure–activity analysis

(2008) N.D. Sonawane et al.   BIOORGANIC & MEDICINAL CHEMISTRY

4′-Methyl-4,5′-bithiazole-based correctors of defective ΔF508-CFTR cellular processing

(2008) Choong Leol Yoo et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Nanomolar CFTR Inhibition by Pore-Occluding Divalent Polyethylene Glycol-Malonic Acid Hydrazides

(2008) N.D. Sonawane et al.   CHEMISTRY & BIOLOGY

Potents-cis-Locked Bithiazole Correctors of ΔF508 Cystic Fibrosis Transmembrane Conductance Regulator Cellular Processing for Cystic Fibrosis Therapy⊥

(2008) Gui Jun Yu et al.   JOURNAL OF MEDICINAL CHEMISTRY

Small-Molecule CFTR Inhibitors Slow Cyst Growth in Polycystic Kidney Disease

(2008) B. Yang et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Disruption of the CFTR Gene Produces a Model of Cystic Fibrosis in Newborn Pigs

(2008) C. S. Rogers et al.   SCIENCE

TMEM16A, A Membrane Protein Associated with Calcium-Dependent Chloride Channel Activity

(2008) A. Caputo et al.   SCIENCE