ArticleNucleic acids research2024
High ionic strength vector formulations enhance gene transfer to airway epithelia.
Article in Nucleic acids research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed.
- Micro/nanoscale bubble-mediated mucolytic conditioning of the lung model of cystic fibrosis enhances nanoparticle transport through the mucus.Biomaterials · 2026Article
- Efficient and safe lung gene delivery using AAV6.2FF in neonatal pigs demonstrates pediatric translational potential.Molecular therapy. Advances · 2026Article
- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- Optimized AAV capsids robustly transduce airway epithelial cells.bioRxiv : the preprint server for biology · 2026Article
- Article
- Porcine airway tissue explant culture at a hydrogel-air interface to model submucosal glands in cystic fibrosis progression and therapy.Science advances · 2025Article
- THERAPIES FOR NEONATAL DISEASES OF THE SURFACTANT SYSTEM.Transactions of the American Clinical and Climatological Association · 2025Review
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6 authors.
Funding
Abstract
A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelia to achieve therapeutic correction. Hypertonic saline (HTS) is frequently administered to people with CF to enhance mucus clearance. HTS transiently disrupts epithelial cell tight junctions, but its ability to improve gene transfer has not been investigated. Here, we asked if increasing the concentration of NaCl enhances the transduction efficiency of three gene therapy vectors: adenovirus, AAV, and lentiviral vectors. Vectors formulated with 3-7% NaCl exhibited markedly increased transduction for all three platforms, leading to anion channel correction in primary cultures of human CF epithelial cells and enhanced gene transfer in mouse and pig airways in vivo. The mechanism of transduction enhancement involved tonicity but not osmolarity or pH. Formulating vectors with a high ionic strength solution is a simple strategy to greatly enhance efficacy and immediately improve preclinical or clinical applications.
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