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Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Nature communications, 2019-08, Vol.10 (1), p.3556-11, Article 3556

2019

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Assays of CFTR Function In Vitro, Ex Vivo and In Vivo

International journal of molecular sciences, 2022-01, Vol.23 (3), p.1437

2022

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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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Author Correction: Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Nature communications, 2020-10, Vol.11 (1), p.5457-5457, Article 5457

2020

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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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Elevated expression of both mRNA and protein levels of IL-17A in sputum of stable Cystic Fibrosis patients

Respiratory research, 2010-12, Vol.11 (1), p.177-177, Article 177

2010

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Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

The New England journal of medicine, 2015-07, Vol.373 (3), p.220-231

2015

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The Effect of Synonymous Single-Nucleotide Polymorphisms on an Atypical Cystic Fibrosis Clinical Presentation

Life (Basel, Switzerland), 2020-12, Vol.11 (1), p.14

2020

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Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell

European Journal of Pediatrics, 2016-01, Vol.175 (1), p.1-8

2016

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A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect

The lancet respiratory medicine, 2013-04, Vol.1 (2), p.158-163

2013

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Cystic fibrosis in the year 2020: A disease with a new face

Acta Paediatrica, 2020-05, Vol.109 (5), p.893-899

De Boeck, Kris

2020

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Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

American journal of respiratory and critical care medicine, 2010-11, Vol.182 (10), p.1262-1272

2010

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Cross-Sectional and Longitudinal Comparisons of the Predominant Fecal Microbiota Compositions of a Group of Pediatric Patients with Cystic Fibrosis and Their Healthy Siblings

Applied and Environmental Microbiology, 2011-11, Vol.77 (22), p.8015-8024

2011

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A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Frontiers in pharmacology, 2021-11, Vol.12, p.746710-746710

2021

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Primary ciliary dyskinesia, an orphan disease

European journal of pediatrics, 2013-02, Vol.172 (2), p.151-162

2013

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Optimisation of children z-score calculation based on new statistical techniques

PloS one, 2018-12, Vol.13 (12), p.e0208362-e0208362

2018

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Mucus Release and Airway Constriction by TMEM16A May Worsen Pathology in Inflammatory Lung Disease

International journal of molecular sciences, 2021-08, Vol.22 (15), p.7852

2021

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Diagnosis of Cystic Fibrosis in Nonscreened Populations

The Journal of pediatrics, 2017-02, Vol.181, p.S52-S57.e2

2017

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Airway tapering: an objective image biomarker for bronchiectasis

European radiology, 2020-05, Vol.30 (5), p.2703-2711

2020

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Association between faecal pH and fat absorption in children with cystic fibrosis on a controlled diet and enzyme supplements dose

Pediatric research, 2021-01, Vol.89 (1), p.205-210

2021

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Assessing gastro-intestinal related quality of life in cystic fibrosis: Validation of PedsQL GI in children and their parents

PloS one, 2019-12, Vol.14 (12), p.e0225004-e0225004

2019

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The effect of CFTR modulators on structural lung disease in cystic fibrosis

Frontiers in pharmacology, 2023-04, Vol.14, p.1147348-1147348

2023

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Diagnostic accuracy of nitric oxide measurements to detect primary ciliary dyskinesia

European journal of clinical investigation, 2014-05, Vol.44 (5), p.477-485

2014

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Defining Polysaccharide Antibody Deficiency: Measurement of Anti-Pneumococcal Antibodies and Anti-Salmonella typhi Antibodies in a Cohort of Patients with Recurrent Infections

Journal of clinical immunology, 2020, Vol.40 (1), p.105-113

2020

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Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Assays of CFTR Function In Vitro, Ex Vivo and In Vivo

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

Author Correction: Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Progress in therapies for cystic fibrosis

Elevated expression of both mRNA and protein levels of IL-17A in sputum of stable Cystic Fibrosis patients

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

The Effect of Synonymous Single-Nucleotide Polymorphisms on an Atypical Cystic Fibrosis Clinical Presentation

Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell

A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect

Cystic fibrosis in the year 2020: A disease with a new face

Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

Cross-Sectional and Longitudinal Comparisons of the Predominant Fecal Microbiota Compositions of a Group of Pediatric Patients with Cystic Fibrosis and Their Healthy Siblings

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Primary ciliary dyskinesia, an orphan disease

Optimisation of children z-score calculation based on new statistical techniques

Mucus Release and Airway Constriction by TMEM16A May Worsen Pathology in Inflammatory Lung Disease

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

Diagnosis of Cystic Fibrosis in Nonscreened Populations

Airway tapering: an objective image biomarker for bronchiectasis

Association between faecal pH and fat absorption in children with cystic fibrosis on a controlled diet and enzyme supplements dose

Assessing gastro-intestinal related quality of life in cystic fibrosis: Validation of PedsQL GI in children and their parents

The effect of CFTR modulators on structural lung disease in cystic fibrosis

Diagnostic accuracy of nitric oxide measurements to detect primary ciliary dyskinesia

Defining Polysaccharide Antibody Deficiency: Measurement of Anti-Pneumococcal Antibodies and Anti-Salmonella typhi Antibodies in a Cohort of Patients with Recurrent Infections

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