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

Nature chemical biology, 2013-07, Vol.9 (7), p.444-454

2013

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Editorial: Advancing therapeutic strategies: exploring ABC transporters and chemicals affecting their expression and function for disease treatment

Frontiers in pharmacology, 2024-05, Vol.15, p.1423979-1423979

2024

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

JCI insight, 2020-09, Vol.5 (18)

2020

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Folding correctors can restore CFTR posttranslational folding landscape by allosteric domain-domain coupling

Nature communications, 2023-10, Vol.14 (1), p.6868-6868, Article 6868

2023

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Mutation-specific downregulation of CFTR2 variants by gating potentiators

Human molecular genetics, 2017-12, Vol.26 (24), p.4873-4885

2017

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Chaperones rescue the energetic landscape of mutant CFTR at single molecule and in cell

Nature communications, 2017-08, Vol.8 (1), p.398-15, Article 398

2017

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Recessive and dominant mutations in COL12A1 cause a novel EDS/myopathy overlap syndrome in humans and mice

Human molecular genetics, 2014-05, Vol.23 (9), p.2339-2352

2014

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

Molecular biology of the cell, 2012-11, Vol.23 (21), p.4188-4202

2012

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Extracellular oxidation in cystic fibrosis airway epithelium causes enhanced EGFR/ADAM17 activity

American journal of physiology. Lung cellular and molecular physiology, 2018-04, Vol.314 (4), p.L555-L568

2018

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Type XII collagen regulates osteoblast polarity and communication during bone formation

The Journal of cell biology, 2011-06, Vol.193 (6), p.1115-1130

2011

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Editorial: Mechanisms of Novel Drugs and Gene Modifiers in the Treatment of Cystic Fibrosis

Frontiers in molecular biosciences, 2022-07, Vol.9, p.975946-975946

2022

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Readthrough-induced misincorporated amino acid ratios guide mutant-specific therapeutic approaches for two CFTR nonsense mutations

Frontiers in pharmacology, 2024-04, Vol.15, p.1389586-1389586

2024

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Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression Defect

PLoS biology, 2016-05, Vol.14 (5), p.e1002462-e1002462

2016

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A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

Journal of personalized medicine, 2021-07, Vol.11 (7), p.643

2021

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Collagen XXVIII, a Novel von Willebrand Factor A Domain-containing Protein with Many Imperfections in the Collagenous Domain

The Journal of biological chemistry, 2006-02, Vol.281 (6), p.3494-3504

2006

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Collagen XII Interacts with Avian Tenascin-X through Its NC3 Domain

The Journal of biological chemistry, 2006-09, Vol.281 (37), p.27461-27470

2006

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Epithelial Anion Transport as Modulator of Chemokine Signaling

Mediators of inflammation, 2016-01, Vol.2016 (2016), p.1-20

2016

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Elexacaftor co-potentiates the activity of F508del and gating mutants of CFTR

Journal of cystic fibrosis, 2021-09, Vol.20 (5), p.895-898

2021

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Discovery of CFTR modulators for the treatment of cystic fibrosis

Expert opinion on drug discovery, 2021-08, Vol.16 (8), p.897-913

2021

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Shedding of Collagen XXIII Is Mediated by Furin and Depends on the Plasma Membrane Microenvironment

The Journal of biological chemistry, 2007-09, Vol.282 (37), p.27424-27435

2007

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Expression of Type XXIII Collagen mRNA and Protein

The Journal of biological chemistry, 2006-07, Vol.281 (30), p.21546-21557

2006

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Synthesis and Biological Evaluation of Pyrazole–Pyrimidones as a New Class of Correctors of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)

Journal of medicinal chemistry, 2024-08, Vol.67 (16), p.13891-13908

2024

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

Nature medicine, 2018-11, Vol.24 (11), p.1732-1742

2018

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Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression

Science translational medicine, 2014-07, Vol.6 (246), p.246ra97

2014

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Analysis of obstetric complications and uterine connective tissue in tenascin-X-deficient humans and mice

Cell and tissue research, 2008-06, Vol.332 (3), p.523-532

2008

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

Editorial: Advancing therapeutic strategies: exploring ABC transporters and chemicals affecting their expression and function for disease treatment

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

Folding correctors can restore CFTR posttranslational folding landscape by allosteric domain-domain coupling

Mutation-specific downregulation of CFTR2 variants by gating potentiators

Chaperones rescue the energetic landscape of mutant CFTR at single molecule and in cell

Recessive and dominant mutations in COL12A1 cause a novel EDS/myopathy overlap syndrome in humans and mice

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

Extracellular oxidation in cystic fibrosis airway epithelium causes enhanced EGFR/ADAM17 activity

Type XII collagen regulates osteoblast polarity and communication during bone formation

Editorial: Mechanisms of Novel Drugs and Gene Modifiers in the Treatment of Cystic Fibrosis

Readthrough-induced misincorporated amino acid ratios guide mutant-specific therapeutic approaches for two CFTR nonsense mutations

Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression Defect

A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

Collagen XXVIII, a Novel von Willebrand Factor A Domain-containing Protein with Many Imperfections in the Collagenous Domain

Collagen XII Interacts with Avian Tenascin-X through Its NC3 Domain

Epithelial Anion Transport as Modulator of Chemokine Signaling

Elexacaftor co-potentiates the activity of F508del and gating mutants of CFTR

Discovery of CFTR modulators for the treatment of cystic fibrosis

Shedding of Collagen XXIII Is Mediated by Furin and Depends on the Plasma Membrane Microenvironment

Expression of Type XXIII Collagen mRNA and Protein

Synthesis and Biological Evaluation of Pyrazole–Pyrimidones as a New Class of Correctors of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)

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

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression

Analysis of obstetric complications and uterine connective tissue in tenascin-X-deficient humans and mice

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