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Progress in therapies for cystic fibrosis

The lancet respiratory medicine, 2016-08, Vol.4 (8), p.662-674

2016

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

Nature genetics, 2013-10, Vol.45 (10), p.1160-1167

2013

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Inhibition of calpain 1 restores plasma membrane stability to pharmacologically rescued Phe508del-CFTR variant

The Journal of biological chemistry, 2019-09, Vol.294 (36), p.13396-13410

2019

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High-Content siRNA Screen Reveals Global ENaC Regulators and Potential Cystic Fibrosis Therapy Targets

Cell, 2013-09, Vol.154 (6), p.1390-1400

2013

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A molecular switch in the scaffold NHERF1 enables misfolded CFTR to evade the peripheral quality control checkpoint

Science signaling, 2015-05, Vol.8 (377), p.ra48

2015

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Transcriptome meta-analysis reveals common differential and global gene expression profiles in cystic fibrosis and other respiratory disorders and identifies CFTR regulators

Genomics (San Diego, Calif.), 2015-11, Vol.106 (5), p.268-277

2015

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Most F508del-CFTR Is Targeted to Degradation at an Early Folding Checkpoint and Independently of Calnexin

Molecular and Cellular Biology, 2005-06, Vol.25 (12), p.5242-5252

2005

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Protocol for Application, Standardization and Validation of the Forskolin-Induced Swelling Assay in Cystic Fibrosis Human Colon Organoids

STAR protocols, 2020-06, Vol.1 (1), p.100019-100019, Article 100019

2020

Volltextzugriff (PDF)

How to determine the mechanism of action of CFTR modulator compounds: A gateway to theranostics

European journal of medicinal chemistry, 2021-01, Vol.210, p.112989-112989, Article 112989

Amaral, Margarida D.

2021

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A central role of the endoplasmic reticulum in the cell emerges from its functional contact sites with multiple organelles

Cellular and molecular life sciences : CMLS, 2020-12, Vol.77 (23), p.4729-4745

2020

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New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls

Pharmacology & therapeutics (Oxford), 2015-01, Vol.145, p.19-34

2015

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Processing of CFTR: Traversing the cellular maze-How much CFTR needs to go through to avoid cystic fibrosis?

Pediatric pulmonology, 2005-06, Vol.39 (6), p.479-491

Amaral, Margarida D.

2005

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Neuronal signaling modulates protein homeostasis in Caenorhabditis elegans post-synaptic muscle cells

Genes & development, 2007-11, Vol.21 (22), p.3006-3016

2007

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Transcription-Dependent Spatial Arrangements of CFTR and Adjacent Genes in Human Cell Nuclei

The Journal of cell biology, 2004-09, Vol.166 (6), p.815-825

2004

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CFTR and chaperones: processing and degradation

Journal of molecular neuroscience, 2004-01, Vol.23 (1-2), p.41-48

Amaral, Margarida D

2004

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Contribution of Casein Kinase 2 and Spleen Tyrosine Kinase to CFTR Trafficking and Protein Kinase A-Induced Activity

Molecular and Cellular Biology, 2011-11, Vol.31 (22), p.4392-4404

2011

Volltextzugriff (PDF)

Letter to the editor of JCF

Journal of cystic fibrosis, 2020-05, Vol.19 (3), p.503-503

Amaral, Margarida D.

2020

Volltextzugriff (PDF)

Antagonistic Regulation of Cystic Fibrosis Transmembrane Conductance Regulator Cell Surface Expression by Protein Kinases WNK4 and Spleen Tyrosine Kinase

Molecular and Cellular Biology, 2011-10, Vol.31 (19), p.4076-4086

2011

Volltextzugriff (PDF)

PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR

European journal of pharmacology, 2024-03, Vol.967, p.176390-176390, Article 176390

2024

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Revertant Mutants G550E and 4RK Rescue Cystic Fibrosis Mutants in the First Nucleotide-Binding domain of CFTR by Different Mechanisms

Proceedings of the National Academy of Sciences - PNAS, 2006-11, Vol.103 (47), p.17891-17896

2006

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Control of cystic fibrosis transmembrane conductance regulator membrane trafficking: not just from the endoplasmic reticulum to the Golgi

The FEBS journal, 2013-09, Vol.280 (18), p.4396-4406

2013

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EPAC1 activation by cAMP stabilizes CFTR at the membrane by promoting its interaction with NHERF1

Journal of cell science, 2016-07, Vol.129 (13), p.2599-2612

2016

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Development of novel therapeutics for all individuals with CF (the future goes on)

Journal of cystic fibrosis, 2023-03, Vol.22, p.S45-S49

2023

Volltextzugriff (PDF)

CFTR mutations altering CFTR fragmentation

Biochemical journal, 2013-01, Vol.449 (1), p.295-305

2013

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Regulation of the Epithelial Na+ Channel by the Protein Kinase CK2

The Journal of biological chemistry, 2008-05, Vol.283 (19), p.13225-13232

2008

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Progress in therapies for cystic fibrosis

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

Inhibition of calpain 1 restores plasma membrane stability to pharmacologically rescued Phe508del-CFTR variant

High-Content siRNA Screen Reveals Global ENaC Regulators and Potential Cystic Fibrosis Therapy Targets

A molecular switch in the scaffold NHERF1 enables misfolded CFTR to evade the peripheral quality control checkpoint

Transcriptome meta-analysis reveals common differential and global gene expression profiles in cystic fibrosis and other respiratory disorders and identifies CFTR regulators

Most F508del-CFTR Is Targeted to Degradation at an Early Folding Checkpoint and Independently of Calnexin

Protocol for Application, Standardization and Validation of the Forskolin-Induced Swelling Assay in Cystic Fibrosis Human Colon Organoids

How to determine the mechanism of action of CFTR modulator compounds: A gateway to theranostics

A central role of the endoplasmic reticulum in the cell emerges from its functional contact sites with multiple organelles

New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls

Processing of CFTR: Traversing the cellular maze-How much CFTR needs to go through to avoid cystic fibrosis?

Neuronal signaling modulates protein homeostasis in Caenorhabditis elegans post-synaptic muscle cells

Transcription-Dependent Spatial Arrangements of CFTR and Adjacent Genes in Human Cell Nuclei

CFTR and chaperones: processing and degradation

Contribution of Casein Kinase 2 and Spleen Tyrosine Kinase to CFTR Trafficking and Protein Kinase A-Induced Activity

Letter to the editor of JCF

Antagonistic Regulation of Cystic Fibrosis Transmembrane Conductance Regulator Cell Surface Expression by Protein Kinases WNK4 and Spleen Tyrosine Kinase

PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR

Revertant Mutants G550E and 4RK Rescue Cystic Fibrosis Mutants in the First Nucleotide-Binding domain of CFTR by Different Mechanisms

Control of cystic fibrosis transmembrane conductance regulator membrane trafficking: not just from the endoplasmic reticulum to the Golgi

EPAC1 activation by cAMP stabilizes CFTR at the membrane by promoting its interaction with NHERF1

Development of novel therapeutics for all individuals with CF (the future goes on)

CFTR mutations altering CFTR fragmentation

Regulation of the Epithelial Na+ Channel by the Protein Kinase CK2

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