ArticleThorax2017
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis.
Article in Thorax, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 88 papers, 2 of them syntheses 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
88 citing papers in PubMed, 2 syntheses or guidelines pooled it, 143 citations in OpenAlex.
- Interventions for preventing distal intestinal obstruction syndrome (DIOS) in cystic fibrosis.The Cochrane database of systematic reviews · 2021Pooled it
- Interventions for preventing distal intestinal obstruction syndrome (DIOS) in cystic fibrosis.The Cochrane database of systematic reviews · 2018Pooled it
- Minimal CBX3-Derived UCOE Confers Long-Term Resistance to Transgene Silencing in Human iPSCs During Neuronal Differentiation.International journal of molecular sciences · 2026Article
- Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice.Gene therapy · 2026Article
- Combining Gene Therapy with Current Modulator Treatments for Cystic Fibrosis: A Promising Area of Research.Pharmaceutics · 2026Review
- Using the nose as a factory to secrete proteins into the lungs or circulation.Molecular therapy. Advances · 2026Article
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- F/HN-pseudotyped lentiviral vector efficiently transduces non-human primate airways with no evidence of relevant toxicity.Molecular therapy. Advances · 2026Article
- Pharmacological and pre-clinical safety profile of rSIV.F/HN, a hybrid lentiviral vector for cystic fibrosis gene therapy.The European respiratory journal · 2025Article
- Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.American journal of respiratory and critical care medicine · 2024Review
- The Road Less Traveled: Slow but Steady Progress Toward Cystic Fibrosis Gene Therapy by the UK Respiratory Gene Therapy Consortium.American journal of respiratory and critical care medicine · 2024Article
- Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models.Gene therapy · 2024Article
- In vivo editing of lung stem cells for durable gene correction in mice.Science (New York, N.Y.) · 2024Article
- Current and future therapeutic approaches of CFTR and airway dysbiosis in an era of personalized medicine.Journal of family medicine and primary care · 2024Review
- Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells.Cell reports. Medicine · 2024Article
- Current landscape of cystic fibrosis gene therapy.Frontiers in pharmacology · 2024Review
- Towards personalized therapies for genetic disorders of surfactant dysfunction.Seminars in fetal & neonatal medicine · 2023Review
- Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Effective viral-mediated lung gene therapy: is airway surface preparation necessary?Gene therapy · 2023Review
- Review
28 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
38 authors at 8 institutions in 5 countries.
Funding
Abstract
We have recently shown that non-viral gene therapy can stabilise the decline of lung function in patients with cystic fibrosis (CF). However, the effect was modest, and more potent gene transfer agents are still required. Fuson protein (F)/Hemagglutinin/Neuraminidase protein (HN)-pseudotyped lentiviral vectors are more efficient for lung gene transfer than non-viral vectors in preclinical models. In preparation for a first-in-man CF trial using the lentiviral vector, we have undertaken key translational preclinical studies. Regulatory-compliant vectors carrying a range of promoter/enhancer elements were assessed in mice and human air-liquid interface (ALI) cultures to select the lead candidate; cystic fibrosis transmembrane conductance receptor (CFTR) expression and function were assessed in CF models using this lead candidate vector. Toxicity was assessed and 'benchmarked' against the leading non-viral formulation recently used in a Phase IIb clinical trial. Integration site profiles were mapped and transduction efficiency determined to inform clinical trial dose-ranging. The impact of pre-existing and acquired immunity against the vector and vector stability in several clinically relevant delivery devices was assessed. A hybrid promoter hybrid cytosine guanine dinucleotide (CpG)- free CMV enhancer/elongation factor 1 alpha promoter (hCEF) consisting of the elongation factor 1α promoter and the cytomegalovirus enhancer was most efficacious in both murine lungs and human ALI cultures (both at least 2-log orders above background). The efficacy (at least 14% of airway cells transduced), toxicity and integration site profile supports further progression towards clinical trial and pre-existing and acquired immune responses do not interfere with vector efficacy. The lead rSIV.F/HN candidate expresses functional CFTR and the vector retains 90-100% transduction efficiency in clinically relevant delivery devices. The data support the progression of the F/HN-pseudotyped lentiviral vector into a first-in-man CF trial in 2017.
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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.