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

Amplifiers co-translationally enhance CFTR biosynthesis via PCBP1-mediated regulation of CFTR mRNA

(2020) Danijela Dukovski et al.   Journal of Cystic Fibrosis

Variable cellular ivacaftor concentrations in people with cystic fibrosis on modulator therapy

(2020) Jennifer S. Guimbellot et al.   Journal of Cystic Fibrosis

CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials

(2019) Scott C. Bell et al.   Journal of Cystic Fibrosis

Mutation-specific dual potentiators maximize rescue of CFTR gating mutants

(2019) Guido Veit et al.   Journal of Cystic Fibrosis

CFTR processing, trafficking and interactions

(2019) Margarida D. Amaral et al.   Journal of Cystic Fibrosis

Biological Characterization of F508delCFTR Protein Processing by the CFTR Corrector ABBV-2222/GLPG2222

(2019) Ashvani K. Singh et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

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

(2019) Harry G M Heijerman et al.   LANCET

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

(2019) Peter G. Middleton et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cystic Fibrosis Lung Disease: An Overview

(2019) Nelson L Turcios   Respiratory Care

Nanomolar-potency ‘co-potentiator’ therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants

(2019) Puay-Wah Phuan et al.   Scientific Reports

Comprehensive mapping of cystic fibrosis mutations to CFTR protein identifies mutation clusters and molecular docking predicts corrector binding site

(2018) Steven V. Molinski et al.   PROTEINS-STRUCTURE FUNCTION AND BIOINFORMATICS

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

(2018) Guido Veit et al.   NATURE MEDICINE

Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response?

(2018) Iwona Pranke et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Dominic Keating et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mutation-specific downregulation of CFTR2 variants by gating potentiators

(2017) Radu G Avramescu et al.   HUMAN MOLECULAR GENETICS

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

Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators

(2017) Iwona M. Pranke et al.   Scientific Reports

β2-Adrenergic receptor agonists activate CFTR in intestinal organoids and subjects with cystic fibrosis

(2016) Lodewijk A.W. Vijftigschild et al.   EUROPEAN RESPIRATORY JOURNAL

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

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

Sustained Benefit from Ivacaftor Demonstrated by Combining Clinical Trial and Cystic Fibrosis Patient Registry Data

(2015) Gregory S. Sawicki et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Pseudomonas aeruginosa in Cystic Fibrosis Patients With G551D-CFTR Treated With Ivacaftor

(2014) Sonya L. Heltshe et al.   CLINICAL INFECTIOUS DISEASES

Cystic fibrosis genetics: from molecular understanding to clinical application

(2014) Garry R. Cutting   NATURE REVIEWS GENETICS

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

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

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

Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene

(2013) Patrick R Sosnay et al.   NATURE GENETICS

Requirements for Efficient Correction of ΔF508 CFTR Revealed by Analyses of Evolved Sequences

(2012) Juan L. Mendoza et al.   CELL

Correction of Both NBD1 Energetics and Domain Interface Is Required to Restore ΔF508 CFTR Folding and Function

(2012) Wael M. Rabeh et al.   CELL

Proinflammatory cytokine secretion is suppressed by TMEM16A or CFTR channel activity in human cystic fibrosis bronchial epithelia

(2012) Guido Veit et al.   MOLECULAR BIOLOGY OF THE CELL

ROCK Inhibitor and Feeder Cells Induce the Conditional Reprogramming of Epithelial Cells

(2011) Xuefeng Liu et al.   AMERICAN JOURNAL OF PATHOLOGY

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

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

Strategies for the Assessment of Protein Aggregates in Pharmaceutical Biotech Product Development

(2010) John den Engelsman et al.   PHARMACEUTICAL RESEARCH

Integrated biophysical studies implicate partial unfolding of NBD1 of CFTR in the molecular pathogenesis of F508del cystic fibrosis

(2010) Chi Wang et al.   PROTEIN SCIENCE

Peripheral Protein Quality Control Removes Unfolded CFTR from the Plasma Membrane

(2010) T. Okiyoneda et al.   SCIENCE

Cooperative Assembly and Misfolding of CFTR Domains In Vivo

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

CFTR Function and Prospects for Therapy

(2008) John R. Riordan   Annual Review of Biochemistry

Solubilizing Mutations Used to Crystallize One CFTR Domain Attenuate the Trafficking and Channel Defects Caused by the Major Cystic Fibrosis Mutation

(2008) Luísa S. Pissarra et al.   CHEMISTRY & BIOLOGY