Nasal Epithelial Cell-Based Models for Individualized Study in Cystic Fibrosis

Rescue of multiple class II CFTR mutations by elexacaftor+ tezacaftor+ivacaftor mediated in part by the dual activities of Elexacaftor as both corrector and potentiator

(2021) Onofrio Laselva et al.   EUROPEAN RESPIRATORY JOURNAL

Significant functional differences in differentiated Conditionally Reprogrammed (CRC)- and Feeder-free Dual SMAD inhibited-expanded human nasal epithelial cells

(2021) Nikhil T. Awatade et al.   Journal of Cystic Fibrosis

Cystic Fibrosis Patients of Minority Race and Ethnicity Less Likely Eligible for CFTR Modulators Based on CFTR Genotype

(2021) Meghan E. McGarry et al.   PEDIATRIC PULMONOLOGY

Amplifiers co-translationally enhance CFTR biosynthesis via PCBP1-mediated regulation of CFTR mRNA

(2020) Danijela Dukovski et al.   Journal of Cystic Fibrosis

A helper-dependent adenoviral vector rescues CFTR to wild type functional levels in CF epithelial cells harbouring class I mutations

(2020) Huibi Cao et al.   EUROPEAN RESPIRATORY JOURNAL

Human Nasal Epithelial Organoids for Therapeutic Development in Cystic Fibrosis

(2020) Zhongyu Liu et al.   Genes

Emerging preclinical modulators developed for F508del-CFTR have the potential to be effective for ORKAMBI resistant processing mutants

(2020) Onofrio Laselva et al.   Journal of Cystic Fibrosis

CFTR modulator therapy for cystic fibrosis caused by the rare c.3700A>G mutation

(2020) Puay-Wah Phuan et al.   Journal of Cystic Fibrosis

Conditionally Reprogrammed Human Normal Airway Epithelial Cells at ALI: A Physiological Model for Emerging Viruses

(2020) Xuefeng Liu et al.   VIROLOGICA SINICA

The CFTR Mutation c.3453G > C (D1152H) Confers an Anion Selectivity Defect in Primary Airway Tissue that Can be Rescued by Ivacaftor

(2020) Onofrio Laselva et al.   Journal of Personalized Medicine

Functional Profiling of CFTR-Directed Therapeutics Using Pediatric Patient-Derived Nasal Epithelial Cell Models

(2020) Jeffrey KiHyun Park et al.   Frontiers in Pediatrics

Choice of Differentiation Media Significantly Impacts Cell Lineage and Response to CFTR Modulators in Fully Differentiated Primary Cultures of Cystic Fibrosis Human Airway Epithelial Cells

(2020) Vinciane Saint-Criq et al.   Cells

Ionocytes and CFTR Chloride Channel Expression in Normal and Cystic Fibrosis Nasal and Bronchial Epithelial Cells

(2020) Paolo Scudieri et al.   Cells

Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination

(2020) Guido Veit et al.   JCI Insight

G970R‐CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect

(2020) Meredith C. Fidler et al.   CTS-Clinical and Translational Science

A Revised Protocol for Culture of Airway Epithelial Cells as a Diagnostic Tool for Primary Ciliary Dyskinesia

(2020) Janice L. Coles et al.   Journal of Clinical Medicine

Decreased mRNA and protein stability of W1282X limits response to modulator therapy

(2019) M.A. Aksit et al.   Journal of Cystic Fibrosis

Two CFTR mutations within codon 970 differently impact on the chloride channel functionality

(2019) Felice Amato et al.   HUMAN MUTATION

Correlating CFTR Function with Clinical Features to Inform Precision Treatment of Cystic Fibrosis

(2019) Allison F McCague et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Structural identification of a hotspot on CFTR for potentiation

(2019) Fangyu Liu et al.   SCIENCE

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

(2019) Yu-Sheng Wu et al.   MOLECULAR PHARMACOLOGY

Intestinal organoids for Cystic Fibrosis research

(2019) E. de Poel et al.   Journal of Cystic Fibrosis

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

(2019) Harry G M Heijerman et al.   LANCET

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

(2019) Peter G. Middleton et al.   NEW ENGLAND JOURNAL OF MEDICINE

Functional rescue of c.3846G>A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of action

(2019) Onofrio Laselva et al.   Journal of Cystic Fibrosis

Personalised CFTR pharmacotherapeutic response testing and therapy of cystic fibrosis

(2018) Cormac McCarthy et al.   EUROPEAN RESPIRATORY JOURNAL

Detection of CFTR function and modulation in primary human nasal cell spheroids

(2018) John J. Brewington et al.   Journal of Cystic Fibrosis

Effectiveness of ivacaftor in cystic fibrosis patients with non- G551D gating mutations

(2018) Jennifer Guimbellot et al.   Journal of Cystic Fibrosis

CFTR modulator theratyping: Current status, gaps and future directions

(2018) John Paul Clancy et al.   Journal of Cystic Fibrosis

Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

(2018) Ema Robinson et al.   MOLECULAR THERAPY

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

(2018) John J. Brewington et al.   Jove-Journal of Visualized Experiments

Structure-guided combination therapy to potently improve the function of mutant CFTRs

(2018) Guido Veit et al.   NATURE MEDICINE

A novel triple combination of pharmacological chaperones improves F508del-CFTR correction

(2018) Graeme W. Carlile et al.   Scientific Reports

Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response?

(2018) Iwona Pranke et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

The effect of premature termination codon mutations on CFTR mRNA abundance in human nasal epithelium and intestinal organoids: a basis for read‐through therapies in cystic fibrosis

(2018) Luka A. Clarke et al.   HUMAN MUTATION

Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis

(2018) Isabelle Sermet-Gaudelus et al.   Journal of Cystic Fibrosis

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Dominic Keating et al.   NEW ENGLAND JOURNAL OF MEDICINE

Therapeutic benefit observed with the CFTR potentiator, ivacaftor, in a CF patient homozygous for the W1282X CFTR nonsense mutation

(2017) Venkateshwar Mutyam et al.   Journal of Cystic Fibrosis

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

(2017) Jennifer L. Taylor-Cousar et al.   NEW ENGLAND JOURNAL OF MEDICINE

Conditional reprogramming of pediatric airway epithelial cells: A new human model to investigate early-life respiratory disorders

(2017) S. Wolf et al.   PEDIATRIC ALLERGY AND IMMUNOLOGY

In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N-of-1 studies

(2017) Meghan E. McGarry et al.   PEDIATRIC PULMONOLOGY

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

Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia

(2017) Saumel Ahmadi et al.   npj Genomic Medicine

Use of a High-Throughput Phenotypic Screening Strategy to Identify Amplifiers, a Novel Pharmacological Class of Small Molecules That Exhibit Functional Synergy with Potentiators and Correctors

(2017) Kenneth A. Giuliano et al.   SLAS Discovery

Airway Progenitor Clone Formation Is Enhanced by Y-27632–Dependent Changes in the Transcriptome

(2016) Susan D. Reynolds et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Correctors and Potentiators Rescue Function of the Truncated W1282X-Cystic Fibrosis Transmembrane Regulator (CFTR) Translation Product

(2016) Peter M. Haggie et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Pharmacological rescue of mutant CFTR protein improves the viscoelastic properties of CF mucus

(2016) Ambra Gianotti 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

Primary airway epithelial cell culture and asthma in children-lessons learnt and yet to come

(2015) Kirsty McLellan et al.   PEDIATRIC PULMONOLOGY

Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial

(2015) Richard B Moss et al.   Lancet Respiratory Medicine

Testing gene therapy vectors in human primary nasal epithelial cultures

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

Reduction of Bacterial Resistance with Inhaled Antibiotics in the Intensive Care Unit

(2014) Lucy B. Palmer et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Cystic fibrosis genetics: from molecular understanding to clinical application

(2014) Garry R. Cutting   NATURE REVIEWS GENETICS

Nasal Epithelial Cells Can Act as a Physiological Surrogate for Paediatric Asthma Studies

(2014) Surendran Thavagnanam et al.   PLoS One

TGF-Beta Downregulation of Distinct Chloride Channels in Cystic Fibrosis-Affected Epithelia

(2014) Hongtao Sun et al.   PLoS One

Sinupret Activates CFTR and TMEM16A-Dependent Transepithelial Chloride Transport and Improves Indicators of Mucociliary Clearance

(2014) Shaoyan Zhang et al.   PLoS One

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

(2013) Patrick R Sosnay et al.   NATURE GENETICS

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

Ivacaftor potentiation of multiple CFTR channels with gating mutations

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

Comparison of Nasal and Bronchial Epithelial Cells Obtained from Patients with COPD

(2012) David M. Comer et al.   PLoS One

ROCK Inhibitor and Feeder Cells Induce the Conditional Reprogramming of Epithelial Cells

(2011) Xuefeng Liu et al.   AMERICAN JOURNAL OF PATHOLOGY

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Influence of cell background on pharmacological rescue of mutant CFTR

(2010) Nicoletta Pedemonte et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

ΔF508 CFTR processing correction and activity in polarized airway and non-airway cell monolayers

(2010) S.M. Rowe et al.   PULMONARY PHARMACOLOGY & THERAPEUTICS

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

Nasal Epithelial Cells as Surrogates for Bronchial Epithelial Cells in Airway Inflammation Studies

(2008) Catherine M. McDougall et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Feasibility of nasal epithelial brushing for the study of airway epithelial functions in CF infants

(2007) Katharina Mosler et al.   Journal of Cystic Fibrosis