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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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What Role Does CFTR Play in Development, Differentiation, Regeneration and Cancer?

International journal of molecular sciences, 2020-04, Vol.21 (9), p.3133

2020

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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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Rescuing mutant CFTR: a multi-task approach to a better outcome in treating cystic fibrosis

Current pharmaceutical design, 2013, Vol.19 (19), p.3497-3508

2013

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A genetic screening strategy identifies novel regulators of the proteostasis network

PLoS genetics, 2011-12, Vol.7 (12), p.e1002438-e1002438

2011

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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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Molecular targeting of CFTR as a therapeutic approach to cystic fibrosis

Trends in pharmacological sciences (Regular ed.), 2007-07, Vol.28 (7), p.334-341

2007

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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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Using the genome to correct the ion transport defect in cystic fibrosis

The Journal of physiology, 2023-05, Vol.601 (9), p.1573-1582

Amaral, Margarida D.

2023

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Impact of KLF4 on Cell Proliferation and Epithelial Differentiation in the Context of Cystic Fibrosis

International journal of molecular sciences, 2020-09, Vol.21 (18), p.6717

2020

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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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Precision medicine for rare diseases: The times they are A-Changin

Current opinion in pharmacology, 2022-04, Vol.63, p.102201-102201, Article 102201

Amaral, Margarida D.

2022

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CyFi-MAP: an interactive pathway-based resource for cystic fibrosis

Scientific reports, 2021-11, Vol.11 (1), p.22223-22223, Article 22223

2021

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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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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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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

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Targeting CFTR: how to treat cystic fibrosis by CFTR-repairing therapies

Current drug targets, 2011-05, Vol.12 (5), p.683-693

Amaral, Margarida D

2011

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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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Enhanced Expression of Human Epididymis Protein 4 (HE4) Reflecting Pro-Inflammatory Status Is Regulated by CFTR in Cystic Fibrosis Bronchial Epithelial Cells

Frontiers in pharmacology, 2021-05, Vol.12, p.592184-592184

2021

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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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Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids

Journal of personalized medicine, 2021-05, Vol.11 (5), p.421

2021

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Cross-talk of inflammatory mediators and airway epithelium reveals the cystic fibrosis transmembrane conductance regulator as a major target

ERJ open research, 2021-10, Vol.7 (4), p.247

2021

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Assessment of Distinct Electrophysiological Parameters in Rectal Biopsies for the Choice of the Best Diagnosis/Prognosis Biomarkers for Cystic Fibrosis

Frontiers in physiology, 2020-12, Vol.11, p.604580-604580

2020

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Progress in therapies for cystic fibrosis

What Role Does CFTR Play in Development, Differentiation, Regeneration and Cancer?

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

Rescuing mutant CFTR: a multi-task approach to a better outcome in treating cystic fibrosis

A genetic screening strategy identifies novel regulators of the proteostasis network

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

Molecular targeting of CFTR as a therapeutic approach to cystic fibrosis

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

Using the genome to correct the ion transport defect in cystic fibrosis

Impact of KLF4 on Cell Proliferation and Epithelial Differentiation in the Context of Cystic Fibrosis

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

Precision medicine for rare diseases: The times they are A-Changin

CyFi-MAP: an interactive pathway-based resource for cystic fibrosis

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

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

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

Targeting CFTR: how to treat cystic fibrosis by CFTR-repairing therapies

Human Epididymis Protein 4: A Novel Serum Inflammatory Biomarker in Cystic Fibrosis

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

Enhanced Expression of Human Epididymis Protein 4 (HE4) Reflecting Pro-Inflammatory Status Is Regulated by CFTR in Cystic Fibrosis Bronchial Epithelial Cells

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

Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids

Cross-talk of inflammatory mediators and airway epithelium reveals the cystic fibrosis transmembrane conductance regulator as a major target

Assessment of Distinct Electrophysiological Parameters in Rectal Biopsies for the Choice of the Best Diagnosis/Prognosis Biomarkers for Cystic Fibrosis

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