ReviewJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2023
Patient-derived cell models for personalized medicine approaches in cystic fibrosis.
Review in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 1 synthesis or guideline pooled it.
- From Pluripotency to Precision: A Comprehensive Review of hPSCs/iPSCs Models in Neuroscience Research.Stem cell reviews and reports · 2026Pooled it
- Targeted Therapy for Restoring CFTR Activity: From Experimental to Clinical Features.International journal of molecular sciences · 2026Review
- Increased low-molecular-weight mucins in muco-obstructive airway disease limitInfection and immunity · 2026Article
- Predictive capacity of paediatric nasal epithelial cells in sequential CFTR modulator therapy.Thorax · 2026Article
- Reimagining human-centric drug development with new approach methodologies.Science (New York, N.Y.) · 2026Review
- Integrating Human Intestinal Organoids into FDA's New Approach Methodologies for Drug Discovery.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Review
- Iloprost-loaded inhalable Nano into Micro (NiM) formulations for treating hyper-inflammation in a pre-clinical model of cystic fibrosis airway epithelial cells.Drug delivery and translational research · 2026Article
- Human organoids as 3D in vitro platforms for drug discovery: opportunities and challenges.Nature reviews. Drug discovery · 2026Review
- Beyond F508del: equity as the next frontier in cystic fibrosis.Lancet regional health. Americas · 2026Article
- Organoid-on-a-chip (OrgOC): Advancing cystic fibrosis research.Materials today. Bio · 2025Review
- Mesenchymal stromal cell extracellular vesicles reduceAmerican journal of physiology. Lung cellular and molecular physiology · 2025Article
- Induced Pluripotent (iPSC) and Mesenchymal (MSC) Stem Cells for In Vitro Disease Modeling and Regenerative Medicine.International journal of molecular sciences · 2025Review
- Let-7b-5p loaded Mesenchymal Stromal Cell Extracellular Vesicles reducebioRxiv : the preprint server for biology · 2025Article
- InhaledProceedings of the National Academy of Sciences of the United States of America · 2025Article
- Review
- Mapping the oxidative landscape in cystic fibrosis: methodological frontiers and application.Frontiers in pharmacology · 2025Review
- CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids.Orphanet journal of rare diseases · 2024Article
- Patient-Derived Microphysiological Systems for Precision Medicine.Advanced healthcare materials · 2024Review
- Article
- L1077P CFTR pathogenic variant function rescue by Elexacaftor-Tezacaftor-Ivacaftor in cystic fibrosis patient-derived air-liquid interface (ALI) cultures and organoids: in vitro guided personalized therapy of non-F508del patients.Respiratory research · 2023Article
Corrections and comments
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Authors and funding
3 authors.
Funding
Abstract
Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) channel that perturb anion transport across the epithelia of the airways and other organs. To treat cystic fibrosis, strategies that target mutant CFTR have been developed such as correctors that rescue folding and enhance transfer of CFTR to the apical membrane, and potentiators that increase CFTR channel activity. While there has been tremendous progress in development and approval of CFTR therapeutics for the most common (F508del) and several other CFTR mutations, around 10-20% of people with cystic fibrosis have rare mutations that are still without an effective treatment. In the current decade, there was an impressive evolution of patient-derived cell models for precision medicine. In cystic fibrosis, these models have played a crucial role in characterizing the molecular defects in CFTR mutants and identifying compounds that target these defects. Cells from nasal, bronchial, and rectal epithelia are most suitable to evaluate treatments that target CFTR. In vitro assays using cultures grown at an air-liquid interface or as organoids and spheroids allow the diagnosis of the CFTR defect and assessment of potential treatment strategies. An overview of currently established cell culture models and assays for personalized medicine approaches in cystic fibrosis will be provided in this review. These models allow theratyping of rare CFTR mutations with available modulator compounds to predict clinical efficacy. Besides evaluation of individual personalized responses to CFTR therapeutics, patient-derived culture models are valuable for testing responses to developmental treatments such as novel RNA- and DNA-based therapies.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.