ReviewFrontiers in pharmacology2022
Gene therapy for cystic fibrosis: Challenges and prospects.
Review in Frontiers in pharmacology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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
19 citing papers in PubMed, 45 citations in OpenAlex.
- F/HN-pseudotyped lentiviral vector efficiently transduces non-human primate airways with no evidence of relevant toxicity.Molecular therapy. Advances · 2026Article
- Soft Artificial Ciliary Brush with Integrated Haptic Feedback for Efficient Airway Mucus Cleaning.Advanced intelligent systems (Weinheim an der Bergstrasse, Germany) · 2026Article
- Neutrophil-derived biomarkers in bronchiectasis: identifying a common therapeutic target.The European respiratory journal · 2025Review
- Review
- Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives.Current genomics · 2025Review
- Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.American journal of respiratory and critical care medicine · 2024Review
- Cystic Fibrosis Modulator Therapies: Bridging Insights from CF to other Membrane Protein Misfolding Diseases.Israel journal of chemistry · 2024Article
- Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models.Gene therapy · 2024Article
- The ageing of people living with cystic fibrosis: what to expect now?European respiratory review : an official journal of the European Respiratory Society · 2024Review
- Placental Gene Therapy for Fetal Growth Restriction and Preeclampsia: Preclinical Studies and Prospects for Clinical Application.Journal of clinical medicine · 2024Review
- Nanomaterials-assisted gene editing and synthetic biology for optimizing the treatment of pulmonary diseases.Journal of nanobiotechnology · 2024Review
- Current and future therapeutic approaches of CFTR and airway dysbiosis in an era of personalized medicine.Journal of family medicine and primary care · 2024Review
- Article
- In Silico and In Vitro Evaluation of the Mechanism of Action of Three VX809-Based Hybrid Derivatives as Correctors of the F508del CFTR Protein.Pharmaceuticals (Basel, Switzerland) · 2023Article
- ATP12A Proton Pump as an Emerging Therapeutic Target in Cystic Fibrosis and Other Respiratory Diseases.Biomolecules · 2023Review
- Examination of Factors Affecting Site-Directed RNA Editing by the MS2-ADAR1 Deaminase System.Genes · 2023Article
- The Role of MMPs in the Era of CFTR Modulators: An Additional Target for Cystic Fibrosis Patients?Biomolecules · 2023Review
- Features of CFTR mRNA and implications for therapeutics development.Frontiers in genetics · 2023Review
- Advanced approaches to overcome biological barriers in respiratory and systemic routes of administration for enhanced nucleic acid delivery to the lung.Expert opinion on drug deliveryReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cystic fibrosis (CF) is a life-threatening autosomal-recessive disease caused by mutations in a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF effects multiple organs, and lung disease is the primary cause of mortality. The median age at death from CF is in the early forties. CF was one of the first diseases to be considered for gene therapy, and efforts focused on treating CF lung disease began shortly after the CFTR gene was identified in 1989. However, despite the quickly established proof-of-concept for
Indexed as
Identifiers
What OpenQuestion holds
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.