Associated Papers
Trikafta restores thermodynamic coupling between two nucleotide binding domains for potentiating CFTR activity.
Wang G
Biomedicine & Pharmacotherapy = Biomedecine & Pharmacotherapie, 2026
Native CFTR codon bias controls translation rate to balance off-pathway aggregation and channel function by conformational imprinting.
Yoon JS et al.
Scientific Reports, 2025
CRISPR for cystic fibrosis: Advances and insights from a systematic review.
Nicosia L et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2025
Thermodynamic coupling between folding correctors and the first of dimerized nucleotide binding domains in CFTR.
Wang G
Research Square, 2025
Effects of Point Mutations on the Thermal Stability of the NBD1 Domain of hCFTR.
Lublin L et al.
Journal of Chemical Information and Modeling, 2025
Global functional genomics reveals GRK5 as a cystic fibrosis therapeutic target synergistic with current modulators.
Botelho HM et al.
Iscience, 2025
Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing.
Carrozzo I et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2025
Unraveling the Mechanism of Action, Binding Sites, and Therapeutic Advances of CFTR Modulators: A Narrative Review.
Baroni D
Current Issues in Molecular Biology, 2025
Allosteric inhibition of CFTR gating by CFTRinh-172 binding in the pore.
Gao X et al.
Nature Communications, 2024
PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR.
Ferreira FC et al.
European Journal of Pharmacology, 2024
A uniquely efficacious type of CFTR corrector with complementary mode of action.
Marchesin V et al.
Science Advances, 2024
Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor.
Ferreira FC et al.
Molecules (basel, Switzerland), 2024
Mutation accumulation in H. sapiens F508del CFTR countermands dN/dS type genomic analysis.
Hong JS et al.
Plos One, 2024
Folding correctors can restore CFTR posttranslational folding landscape by allosteric domain-domain coupling.
Soya N et al.
Nature Communications, 2023
Identification of novel F508del-CFTR traffic correctors among triazole derivatives.
Bacalhau M et al.
European Journal of Pharmacology, 2023
Pharmacological chaperone-rescued cystic fibrosis CFTR-F508del mutant overcomes PRAF2-gated access to endoplasmic reticulum exit sites.
Saha K et al.
Cellular and Molecular Life Sciences : Cmls, 2022
A small molecule CFTR potentiator restores ATP-dependent channel gating to the cystic fibrosis mutant G551D-CFTR.
Liu J et al.
British Journal of Pharmacology, 2022
Structural Comparative Modeling of Multi-Domain F508del CFTR.
McDonald EF et al.
Biomolecules, 2022
Can two wrongs make a right? F508del-CFTR ion channel rescue by second-site mutations in its transmembrane domains.
Prins S et al.
The Journal of Biological Chemistry, 2022
Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A.
Lopes-Pacheco M et al.
Cells, 2022
Rare Trafficking CFTR Mutations Involve Distinct Cellular Retention Machineries and Require Different Rescuing Strategies.
Ramalho SS et al.
International Journal of Molecular Sciences, 2021
Stability Prediction for Mutations in the Cytosolic Domains of Cystic Fibrosis Transmembrane Conductance Regulator.
Bahia MS et al.
Journal of Chemical Information and Modeling, 2021
Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination.
Veit G et al.
Jci Insight, 2020
Biological Characterization of F508delCFTR Protein Processing by the CFTR Corrector ABBV-2222/GLPG2222.
Singh AK et al.
The Journal of Pharmacology and Experimental Therapeutics, 2020
Disease-relevant mutations alter amino acid co-evolution networks in the second nucleotide binding domain of CFTR.
Ivey G et al.
Plos One, 2020
Slowing ribosome velocity restores folding and function of mutant CFTR.
Oliver KE et al.
The Journal of Clinical Investigation, 2019
High-Throughput Surface Liquid Absorption and Secretion Assays to Identify F508del CFTR Correctors Using Patient Primary Airway Epithelial Cultures.
Berg A et al.
Slas Discovery : Advancing Life Sciences R & D, 2019
Folding Status Is Determinant over Traffic-Competence in Defining CFTR Interactors in the Endoplasmic Reticulum.
Santos JD et al.
Cells, 2019
A defective flexible loop contributes to the processing and gating defects of the predominant cystic fibrosis-causing mutation.
Chen X et al.
Faseb Journal : Official Publication of the Federation of American Societies for Experimental Biology, 2019
A pathogenic role for cystic fibrosis transmembrane conductance regulator in celiac disease.
Villella VR et al.
The Embo Journal, 2019
STRUCTURE, GATING, AND REGULATION OF THE CFTR ANION CHANNEL.
Csanády L et al.
Physiological Reviews, 2019
Differential Scanning Fluorimetry and Hydrogen Deuterium Exchange Mass Spectrometry to Monitor the Conformational Dynamics of NBD1 in Cystic Fibrosis.
Soya N et al.
Methods in Molecular Biology (clifton, N.j.), 2019
Structure-guided combination therapy to potently improve the function of mutant CFTRs.
Veit G et al.
Nature Medicine, 2018
ΔF508-CFTR Modulator Screen Based on Cell Surface Targeting of a Chimeric Nucleotide Binding Domain 1 Reporter.
Phuan PW et al.
Slas Discovery : Advancing Life Sciences R & D, 2018
Structural stability of purified human CFTR is systematically improved by mutations in nucleotide binding domain 1.
Yang Z et al.
Biochimica Et Biophysica Acta. Biomembranes, 2018
Insights into channel dysfunction from modelling and molecular dynamics simulations.
Musgaard M et al.
Neuropharmacology, 2018
Substitution of Yor1p NBD1 residues improves the thermal stability of Human Cystic Fibrosis Transmembrane Conductance Regulator.
Xavier BM et al.
Protein Engineering, Design & Selection : Peds, 2017
Chaperones rescue the energetic landscape of mutant CFTR at single molecule and in cell.
Bagdany M et al.
Nature Communications, 2017
From the endoplasmic reticulum to the plasma membrane: mechanisms of CFTR folding and trafficking.
Farinha CM et al.
Cellular and Molecular Life Sciences : Cmls, 2017
Molecular modelling and molecular dynamics of CFTR.
Callebaut I et al.
Cellular and Molecular Life Sciences : Cmls, 2017
Thermal unfolding simulations of NBD1 domain variants reveal structural motifs associated with the impaired folding of F508del-CFTR.
Estácio SG et al.
Molecular Biosystems, 2016
Obligate coupling of CFTR pore opening to tight nucleotide-binding domain dimerization.
Mihályi C et al.
Elife, 2016
Non-native Conformers of Cystic Fibrosis Transmembrane Conductance Regulator NBD1 Are Recognized by Hsp27 and Conjugated to SUMO-2 for Degradation.
Gong X et al.
The Journal of Biological Chemistry, 2016
Deletion of Phenylalanine 508 in the First Nucleotide-binding Domain of the Cystic Fibrosis Transmembrane Conductance Regulator Increases Conformational Exchange and Inhibits Dimerization.
Chong PA et al.
The Journal of Biological Chemistry, 2015
Restoration of NBD1 thermal stability is necessary and sufficient to correct ∆F508 CFTR folding and assembly.
He L et al.
Journal of Molecular Biology, 2015