ReviewHuman gene therapy. Clinical development2015
Ferret and pig models of cystic fibrosis: prospects and promise for gene therapy.
Review in Human gene therapy. Clinical development, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 49 papers.
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
49 citing papers in PubMed, 76 citations in OpenAlex.
- Unravelling the developmental origins of cystic fibrosis-related diabetes.Diabetologia · 2026Review
- Development of a large porcine model of osteogenesis imperfecta type I.Bone reports · 2026Article
- Cystic Fibrosis of the Pancreas: In Vitro Duct Models for CFTR-Targeted Translational Research.International journal of molecular sciences · 2026Review
- A ferret model demonstrates sustained fibrosis, restrictive physiology, and airway proximalization.NPJ Regenerative medicine · 2025Article
- Pulmonary Fibrosis-Focusing on the Future: Aspen Lung Conference 2024 Summary.American journal of respiratory cell and molecular biology · 2025Article
- Isolation and Culture of Ferret Airway Stem Cells.Bio-protocol · 2025Article
- A chronicInfection and immunity · 2025Article
- Comparative anatomy of respiratory bronchioles and lobular structures in mammals.Journal of toxicologic pathology · 2025Review
- Strategies for Modifying Adenoviral Vectors for Gene Therapy.International journal of molecular sciences · 2024Review
- Endocrine pathology in young rabbits with cystic fibrosis.eGastroenterology · 2024Article
- Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs.Molecular therapy. Methods & clinical development · 2024Article
- BMI1 Transduction of Human Airway Epithelial Cells for Expansion of Proliferation and Differentiation.Methods in molecular biology (Clifton, N.J.) · 2024Article
- Effective viral-mediated lung gene therapy: is airway surface preparation necessary?Gene therapy · 2023Review
- Development and characterization of ferretFrontiers in medicine · 2023Article
- Recombinant Adeno-Associated Virus-Mediated Editing of the G551D Cystic Fibrosis Transmembrane Conductance Regulator Mutation in Ferret Airway Basal Cells.Human gene therapy · 2022Article
- The Fingerprints of Biomedical Science in Internal Medicine.Advances in experimental medicine and biology · 2022Article
- Gene therapy for cystic fibrosis: Challenges and prospects.Frontiers in pharmacology · 2022Review
- The use of adenoviral vectors in gene therapy and vaccine approaches.Genetics and molecular biology · 2022Article
- Use of Translational, Genetically Modified Porcine Models to Ultimately Improve Intestinal Disease Treatment.Frontiers in veterinary science · 2022Review
- Transduction of Pig Small Airway Epithelial Cells and Distal Lung Progenitor Cells by AAV4.Cells · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 3 institutions in 2 countries.
Funding
Abstract
Large animal models of genetic diseases are rapidly becoming integral to biomedical research as technologies to manipulate the mammalian genome improve. The creation of cystic fibrosis (CF) ferrets and pigs is an example of such progress in animal modeling, with the disease phenotypes in the ferret and pig models more reflective of human CF disease than mouse models. The ferret and pig CF models also provide unique opportunities to develop and assess the effectiveness of gene and cell therapies to treat affected organs. In this review, we examine the organ disease phenotypes in these new CF models and the opportunities to test gene therapies at various stages of disease progression in affected organs. We then discuss the progress in developing recombinant replication-defective adenoviral, adeno-associated viral, and lentiviral vectors to target genes to the lung and pancreas in ferrets and pigs, the two most affected organs in CF. Through this review, we hope to convey the potential of these new animal models for developing CF gene and cell 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.