Variable Responses to CFTR Correctors in vitro: Estimating the Design Effect in Precision Medicine

Cystic fibrosis transmembrane conductance regulator modulators

(2018) Elizabeth B. Burgener et al.   CURRENT OPINION IN PEDIATRICS

Personalised CFTR pharmacotherapeutic response testing and therapy of cystic fibrosis

(2018) Cormac McCarthy et al.   EUROPEAN RESPIRATORY JOURNAL

Recent progress in translational cystic fibrosis research using precision medicine strategies

(2018) Deborah M. Cholon et al.   Journal of Cystic Fibrosis

Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators

(2017) Iwona M. Pranke et al.   Scientific Reports

Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor)

(2016) Elizabeth Matthes et al.   BRITISH JOURNAL OF PHARMACOLOGY

Cystic fibrosis

(2016) Stacey L. Martiniano et al.   CURRENT OPINION IN PEDIATRICS

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

(2016) Johanna F. Dekkers et al.   Science Translational Medicine

Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients

(2015) M. D. Amaral   JOURNAL OF INTERNAL MEDICINE

Genome-wide association meta-analysis identifies five modifier loci of lung disease severity in cystic fibrosis

(2015) Harriet Corvol et al.   Nature Communications

Cystic fibrosis

(2015) Felix Ratjen et al.   Nature Reviews Disease Primers

VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface

(2014) Paul D.W. Eckford et al.   CHEMISTRY & BIOLOGY

A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial

(2014) Michael P Boyle et al.   Lancet Respiratory Medicine

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

(2012) Patrick A. Flume et al.   CHEST

Multiple apical plasma membrane constituents are associated with susceptibility to meconium ileus in individuals with cystic fibrosis

(2012) Lei Sun et al.   NATURE GENETICS

Mouse models of cystic fibrosis: Phenotypic analysis and research applications

(2011) Martina Wilke et al.   Journal of Cystic Fibrosis

Genome-wide association and linkage identify modifier loci of lung disease severity in cystic fibrosis at 11p13 and 20q13.2

(2011) Fred A Wright et al.   NATURE GENETICS

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Correction of the  Phe508 Cystic Fibrosis Transmembrane Conductance Regulator Trafficking Defect by the Bioavailable Compound Glafenine

(2010) R. Robert et al.   MOLECULAR PHARMACOLOGY

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

(2010) Frank J. Accurso et al.   NEW ENGLAND JOURNAL OF MEDICINE

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CFTR Function and Prospects for Therapy

(2008) John R. Riordan   Annual Review of Biochemistry