A new platform for high-throughput therapy testing on iPSC-derived lung progenitor cells from cystic fibrosis patients

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

Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids

(2021) Daniel K. Crawford et al.   Journal of Cystic Fibrosis

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

(2021) Duncan Keegan et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

(2021) Iris A. L. Silva et al.   Journal of Personalized Medicine

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

The preclinical discovery and development of the combination of ivacaftor + tezacaftor used to treat cystic fibrosis

(2020) Lorenzo Guerra et al.   Expert Opinion on Drug Discovery

Comparison of Organoid Swelling and in vivoBiomarkers of CFTR Function to Determine Effects of Lumacaftor-ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation

(2020) Simon Y. Graeber et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

A Single-cell Atlas of the Human Healthy Airways

(2020) Marie Deprez et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Correction of CFTR function in intestinal organoids to guide treatment of Cystic Fibrosis

(2020) Anabela S. Ramalho et al.   EUROPEAN RESPIRATORY JOURNAL

A functional genomics approach to investigate the differentiation of iPSCs into lung epithelium at air‐liquid interface

(2020) Jenny L. Kerschner et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

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

Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing

(2020) Steven Erwood et al.   Molecular Therapy-Methods & Clinical Development

Derivation of Airway Basal Stem Cells from Human Pluripotent Stem Cells

(2020) Finn J. Hawkins et al.   Cell Stem Cell

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

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

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

(2020) Guido Veit et al.   JCI Insight

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

(2019) Gitte Berkers et al.   Cell Reports

Nonsense Mediated RNA Decay Pathway Inhibition Restores Expression and Function of W1282X CFTR

(2019) Melissa M Keenan et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Identification of GLPG/ABBV-2737, a Novel Class of Corrector, Which Exerts Functional Synergy With Other CFTR Modulators

(2019) Gert de Wilde et al.   Frontiers in Pharmacology

High-Throughput Screening for Modulators of CFTR Activity Based on Genetically Engineered Cystic Fibrosis Disease-Specific iPSCs

(2019) Sylvia Merkert et al.   Stem Cell Reports

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

Forskolin-induced swelling of intestinal organoids correlates with disease severity in adults with cystic fibrosis and homozygous F508del mutations

(2019) Karin M. de Winter – de Groot 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

Comparison of ex vivo and in vitro intestinal cystic fibrosis models to measure CFTR-dependent ion channel activity

(2018) Domenique D. Zomer-van Ommen et al.   Journal of Cystic Fibrosis

Combination potentiator (‘co-potentiator’) therapy for CF caused by CFTR mutants, including N1303K, that are poorly responsive to single potentiators

(2018) Puay-Wah Phuan et al.   Journal of Cystic Fibrosis

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

(2018) Guido Veit et al.   NATURE MEDICINE

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

Isogenic cell models of cystic fibrosis-causing variants in natively expressing pulmonary epithelial cells

(2018) Hillary C. Valley et al.   Journal of Cystic Fibrosis

The CF Canada-Sick Kids Program in individual CF therapy: A resource for the advancement of personalized medicine in CF

(2018) Paul D.W. Eckford et al.   Journal of Cystic Fibrosis

Prospective isolation of NKX2-1–expressing human lung progenitors derived from pluripotent stem cells

(2017) Finn Hawkins et al.   JOURNAL OF CLINICAL INVESTIGATION

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

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

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

Human embryonic lung epithelial tips are multipotent progenitors that can be expanded in vitro as long-term self-renewing organoids

(2017) Marko Z Nikolić et al.   eLife

Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia

(2017) Saumel Ahmadi et al.   npj Genomic Medicine

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

Efficient generation of functional CFTR-expressing airway epithelial cells from human pluripotent stem cells

(2015) Amy P Wong et al.   Nature Protocols

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

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

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

(2015) Ana M. Crane et al.   Stem Cell Reports

Generation of multiciliated cells in functional airway epithelia from human induced pluripotent stem cells

(2014) A. L. Firth et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient generation of lung and airway epithelial cells from human pluripotent stem cells

(2013) Sarah X L Huang et al.   NATURE BIOTECHNOLOGY

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Directed differentiation of human pluripotent stem cells into mature airway epithelia expressing functional CFTR protein

(2012) Amy P Wong et al.   NATURE BIOTECHNOLOGY

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

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

(2011) Gergely L. Lukacs et al.   TRENDS IN MOLECULAR MEDICINE