ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
Adenoviral gene transfer corrects the ion transport defect in the sinus epithelia of a porcine CF model.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.
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Who cites it
16 citing papers in PubMed, 1 synthesis or guideline pooled it, 26 citations in OpenAlex.
- Assessing the consistency of iPSC and animal models in cystic fibrosis modelling: A meta-analysis.PloS one · 2022Pooled it
- Pharmacological and pre-clinical safety profile of rSIV.F/HN, a hybrid lentiviral vector for cystic fibrosis gene therapy.The European respiratory journal · 2025Article
- Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination.Science translational medicine · 2023Article
- Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium.Advanced materials (Deerfield Beach, Fla.) · 2019Article
- Restoration of CFTR Activity in Ducts Rescues Acinar Cell Function and Reduces Inflammation in Pancreatic and Salivary Glands of Mice.Gastroenterology · 2017Article
- Partial Restoration of CFTR Function in cftr-Null Mice following Targeted Cell Replacement Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Lentiviral-mediated phenotypic correction of cystic fibrosis pigs.JCI insight · 2016Article
- Genome editing revolutionize the creation of genetically modified pigs for modeling human diseases.Human genetics · 2016Review
- Lessons learned from the cystic fibrosis pig.Theriogenology · 2016Review
- Relationships among CFTR expression, HCO3- secretion, and host defense may inform gene- and cell-based cystic fibrosis therapies.Proceedings of the National Academy of Sciences of the United States of America · 2016Article
- Cystic Fibrosis Gene Therapy in the UK and Elsewhere.Human gene therapy · 2015Review
- Contemporary Animal Models For Human Gene Therapy Applications.Current gene therapy · 2015Review
- Transduction of ferret airway epithelia using a pre-treatment and lentiviral gene vector.BMC pulmonary medicine · 2014Article
- Porcine nasal epithelial cultures for studies of cystic fibrosis sinusitis.International forum of allergy & rhinology · 2014Article
- New insights into the pathogenesis of cystic fibrosis sinusitis.International forum of allergy & rhinology · 2014Review
- Pulmonary Delivery of Nonviral Nucleic Acid-Based Vaccines With Spotlight on Gold Nanoparticles.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnologyReview
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
Cystic fibrosis (CF) pigs spontaneously develop sinus and lung disease resembling human CF. The CF pig presents a unique opportunity to use gene transfer to test hypotheses to further understand the pathogenesis of CF sinus disease. In this study, we investigated the ion transport defect in the CF sinus and found that CF porcine sinus epithelia lack cyclic AMP (cAMP)-stimulated anion transport. We asked whether we could restore CF transmembrane conductance regulator gene (CFTR) current in the porcine CF sinus epithelia by gene transfer. We quantified CFTR transduction using an adenovirus expressing CFTR and green fluorescent protein (GFP). We found that as little as 7% of transduced cells restored 6% of CFTR current with 17-28% of transduced cells increasing CFTR current to 50% of non-CF levels. We also found that we could overcorrect cAMP-mediated current in non-CF epithelia. Our findings indicate that CF porcine sinus epithelia lack anion transport, and a relatively small number of cells expressing CFTR are required to rescue the ion transport phenotype. These studies support the use of the CF pig as a preclinical model for future gene therapy trials in CF sinusitis.
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