ORKAMBI-Mediated Rescue of Mucociliary Clearance in Cystic Fibrosis Primary Respiratory Cultures Is Enhanced by Arginine Uptake, Arginase Inhibition, and Promotion of Nitric Oxide Signaling to the Cystic Fibrosis Transmembrane Conductance Regulator Channel

Transducing Airway Basal Cells with a Helper-Dependent Adenoviral Vector for Lung Gene Therapy

(2018) Huibi Cao et al.   HUMAN GENE THERAPY

Correctors of the Major Cystic Fibrosis Mutant Interact through Membrane-Spanning Domains

(2018) Onofrio Laselva et al.   MOLECULAR PHARMACOLOGY

Comprehensive mapping of cystic fibrosis mutations to CFTR protein identifies mutation clusters and molecular docking predicts corrector binding site

(2018) Steven V. Molinski et al.   PROTEINS-STRUCTURE FUNCTION AND BIOINFORMATICS

SLC6A14, an amino acid transporter, modifies the primary CF defect in fluid secretion

(2018) Saumel Ahmadi et al.   eLife

Molecular Mechanism of Action of Trimethylangelicin Derivatives as CFTR Modulators

(2018) Onofrio Laselva et al.   Frontiers in Pharmacology

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

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR : a randomised, placebo-controlled phase 3 trial

(2017) Felix Ratjen et al.   Lancet Respiratory Medicine

Inhibition of arginase by CB-1158 blocks myeloid cell-mediated immune suppression in the tumor microenvironment

(2017) Susanne M. Steggerda et al.   Journal for ImmunoTherapy of Cancer

Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia

(2017) Saumel Ahmadi et al.   npj Genomic Medicine

Attenuation of Phosphorylation-dependent Activation of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) by Disease-causing Mutations at the Transmission Interface

(2016) Stephanie Chin et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

A Low-Cost Method of Ciliary Beat Frequency Measurement Using iPhone and MATLAB

(2016) Jason J. Chen et al.   OTOLARYNGOLOGY-HEAD AND NECK SURGERY

Individualized medicine using intestinal responses to CFTR potentiators and correctors

(2016) Jeffrey M. Beekman   PEDIATRIC PULMONOLOGY

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

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

Pharmacokinetics and tolerability of oral sildenafil in adults with cystic fibrosis lung disease

(2015) J.L. Taylor-Cousar et al.   Journal of Cystic Fibrosis

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

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

Testing gene therapy vectors in human primary nasal epithelial cultures

(2015) Huibi Cao et al.   Molecular Therapy-Methods & Clinical Development

Lung arginase expression and activity is increased in cystic fibrosis mouse models

(2014) Thomas Jaecklin et al.   JOURNAL OF APPLIED PHYSIOLOGY

A randomized controlled trial of inhaled l-Arginine in patients with cystic fibrosis

(2013) H. Grasemann et al.   Journal of Cystic Fibrosis

Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function

(2013) Fredrick Van Goor et al.   Journal of Cystic Fibrosis

Arginine Metabolism in Patients with Cystic Fibrosis

(2013) Hartmut Grasemann et al.   JOURNAL OF PEDIATRICS

Vx-770 potentiates CFTR function by promoting decoupling between the gating cycle and ATP hydrolysis cycle

(2013) Kang-Yang Jih et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Development of primary human nasal epithelial cell cultures for the study of cystic fibrosis pathophysiology

(2012) F. de Courcey et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Nitric Oxide and L-Arginine Deficiency in Cystic Fibrosis

(2012) Hartmut Grasemann et al.   CURRENT PHARMACEUTICAL DESIGN

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a Phosphorylation-dependent but ATP-independent Manner

(2012) Paul D. W. Eckford et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Ivacaftor potentiation of multiple CFTR channels with gating mutations

(2012) Haihui Yu et al.   Journal of Cystic Fibrosis

Regulation of ciliary motility: Conserved protein kinases and phosphatases are targeted and anchored in the ciliary axoneme

(2011) Maureen Wirschell et al.   ARCHIVES OF BIOCHEMISTRY AND BIOPHYSICS

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

VX-770, a CFTR potentiator, may have a potential clinical benefit in a subgroup of people with cystic fibrosis

(2011) S. Shah   THORAX

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

Effect of growth hormone therapy on nitric oxide formation in cystic fibrosis patients

(2007) C. Grasemann et al.   EUROPEAN RESPIRATORY JOURNAL