High-throughput functional assay in cystic fibrosis patient-derived organoids allows drug repurposing

Forskolin-induced Organoid Swelling is Associated with Long-term CF Disease Progression

(2022) Danya Muilwijk et al.   EUROPEAN RESPIRATORY JOURNAL

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

Discovery of CFTR modulators for the treatment of cystic fibrosis

(2021) Miquéias Lopes-Pacheco et al.   Expert Opinion on Drug Discovery

Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids

(2021) E. de Poel et al.   Journal of Cystic Fibrosis

A role for AKT1 in nonsense-mediated mRNA decay

(2021) Martine Palma et al.   NUCLEIC ACIDS RESEARCH

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

(2021) Jia Xin Jiang et al.   Stem Cell Reports

PHD1 controls muscle mTORC1 in a hydroxylation-independent manner by stabilizing leucyl tRNA synthetase

(2020) Gommaar D’Hulst et al.   Nature Communications

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

(2019) Gitte Berkers et al.   Cell Reports

High-Throughput Surface Liquid Absorption and Secretion Assays to Identify F508del CFTR Correctors Using Patient Primary Airway Epithelial Cultures

(2019) Allison Berg et al.   SLAS Discovery

Cholesterol Interaction Directly Enhances Intrinsic Activity of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)

(2019) Chin et al.   Cells

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

A Living Biobank of Breast Cancer Organoids Captures Disease Heterogeneity

(2018) Norman Sachs et al.   CELL

Intestinal Failure and Aberrant Lipid Metabolism in Patients With DGAT1 Deficiency

(2018) Jorik M. van Rijn et al.   GASTROENTEROLOGY

Stratifying infants with cystic fibrosis for disease severity using intestinal organoid swelling as a biomarker of CFTR function

(2018) Karin M. de Winter-de Groot et al.   EUROPEAN RESPIRATORY JOURNAL

In situ click chemistry generation of cyclooxygenase-2 inhibitors

(2017) Atul Bhardwaj et al.   Nature Communications

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

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

The AKT/mTOR signaling pathway plays a key role in statin-induced myotoxicity

(2015) Annalisa Bonifacio et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Different effects of statins on induction of diabetes mellitus: an experimental study

(2015) Shui-Ping Zhao et al.   Drug Design Development and Therapy

Cystic fibrosis: An inherited disease affecting mucin-producing organs

(2014) Camille Ehre et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Ex vivo culture of intestinal crypt organoids as a model system for assessing cell death induction in intestinal epithelial cells and enteropathy

(2014) T Grabinger et al.   Cell Death & Disease

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

(2014) Eitan Kerem et al.   Lancet Respiratory Medicine

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

(2013) Patrick R Sosnay et al.   NATURE GENETICS

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Identification of Synergistic Combinations of F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators

(2010) Stephen Lin et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations

(2009) Igor Nudelman et al.   JOURNAL OF MEDICINAL CHEMISTRY