ReviewEuropean respiratory review : an official journal of the European Respiratory Society2013
Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis.
Review in European respiratory review : an official journal of the European Respiratory Society, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
44 citing papers in PubMed, 1 synthesis or guideline pooled it, 121 citations in OpenAlex.
- Cystic Fibrosis-Related Gut Dysbiosis: A Systematic Review.Digestive diseases and sciences · 2023Pooled it
- Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR.American journal of respiratory and critical care medicine · 2018Trial
- Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR.Annals of the American Thoracic Society · 2017Trial
- Elexacaftor/tezacaftor/ivacaftor (ETI) therapy modifies the expression of interferon beta and inflammatory genes in the airways of adult patients with cystic fibrosis: a pilot study.Molecular and cellular biochemistry · 2025Article
- Intestinal luminal anion transporters and their interplay with gut microbiome and inflammation.American journal of physiology. Cell physiology · 2025Review
- Tuning Lipid Nanoparticles for RNA Delivery to Extrahepatic Organs.Advanced materials (Deerfield Beach, Fla.) · 2024Review
- A New Frontier in Cystic Fibrosis Pathophysiology: How and When Clock Genes Can Affect the Inflammatory/Immune Response in a Genetic Disease Model.Current issues in molecular biology · 2024Review
- Pharmacokinetic variability of CFTR modulators from standard and alternative regimens.Pulmonary pharmacology & therapeutics · 2024Article
- Cystic Fibrosis: Understanding Cystic Fibrosis Transmembrane Regulator Mutation Classification and Modulator Therapies.Advances in respiratory medicine · 2024Review
- Patient-Derived Microphysiological Systems for Precision Medicine.Advanced healthcare materials · 2024Review
- Morphological changes in intraepithelial and stromal telocytes in Cyprinus carpio in response to salinity stress.Scientific reports · 2023Article
- Sputum from People with Cystic Fibrosis Reduces the Killing of Methicillin-ResistantPathogens (Basel, Switzerland) · 2023Article
- Plasma and cellular ivacaftor concentrations in patients with cystic fibrosis.Pediatric pulmonology · 2022Article
- CFTR function and clinical response to modulators parallel nasal epithelial organoid swelling.American journal of physiology. Lung cellular and molecular physiology · 2021Article
- ERS International Congress 2020: highlights from the Thoracic Surgery and Transplantation Assembly.ERJ open research · 2021Article
- An Adverse Outcome Pathway for Decreased Lung Function Focusing on Mechanisms of Impaired Mucociliary Clearance Following Inhalation Exposure.Frontiers in toxicology · 2021Article
- Cystic Fibrosis: Systems Biology Analysis from Homozygous p.Phe508del Variant Patients' Samples Reveals Perturbations in Tissue-Specific Pathways.BioMed research international · 2021Article
- Proteomics and Metabolomics for Cystic Fibrosis Research.International journal of molecular sciences · 2020Review
- Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy: A Review for the Otolaryngologist.American journal of rhinology & allergy · 2020Review
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cystic fibrosis (CF) is caused by genetic mutations that affect the cystic fibrosis transmembrane conductance regulator (CFTR) protein. These mutations can impact the synthesis and transfer of the CFTR protein to the apical membrane of epithelial cells, as well as influencing the gating or conductance of chloride and bicarbonate ions through the channel. CFTR dysfunction results in ionic imbalance of epithelial secretions in several organ systems, such as the pancreas, gastrointestinal tract, liver and the respiratory system. Since discovery of the CFTR gene in 1989, research has focussed on targeting the underlying genetic defect to identify a disease-modifying treatment for CF. Investigated management strategies have included gene therapy and the development of small molecules that target CFTR mutations, known as CFTR modulators. CFTR modulators are typically identified by high-throughput screening assays, followed by preclinical validation using cell culture systems. Recently, one such modulator, the CFTR potentiator ivacaftor, was approved as an oral therapy for CF patients with the G551D-CFTR mutation. The clinical development of ivacaftor not only represents a breakthrough in CF care but also serves as a noteworthy example of personalised medicine.
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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.