ArticleGene therapy2011
Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung.
Article in Gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 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
34 citing papers in PubMed, 103 citations in OpenAlex.
- A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial.American journal of respiratory and critical care medicine · 2015Trial
- Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.The Lancet. Respiratory medicine · 2015Trial
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- Impact of nebulizers on nanoparticles-based gene delivery efficiency:Drug delivery · 2025Article
- Delivery of nucleic acids using nanomaterials.Molecular biomedicine · 2023Review
- RNA Nanomedicine: Delivery Strategies and Applications.The AAPS journal · 2023Review
- GL67 lipid-based liposomal formulation for efficient siRNA delivery into human lung cancer cells.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2023Article
- Aerosol-Mediated Non-Viral Lung Gene Therapy: The Potential of Aminoglycoside-Based Cationic Liposomes.Pharmaceutics · 2021Article
- Advanced human-relevant in vitro pulmonary platforms for respiratory therapeutics.Advanced drug delivery reviews · 2021Review
- Recent applications and strategies in nanotechnology for lung diseases.Nano research · 2021Review
- Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia.Nature communications · 2019Article
- Tetrafunctional Block Copolymers Promote Lung Gene Transfer in Newborn Piglets.Molecular therapy. Nucleic acids · 2019Article
- Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium.Advanced materials (Deerfield Beach, Fla.) · 2019Article
- Barriers to inhaled gene therapy of obstructive lung diseases: A review.Journal of controlled release : official journal of the Controlled Release Society · 2016Review
- A novel translational model for fetoscopic intratracheal delivery of nanoparticles in piglets.Prenatal diagnosis · 2016Article
- New and emerging targeted therapies for cystic fibrosis.BMJ (Clinical research ed.) · 2016Review
- Engineering large animal models of human disease.The Journal of pathology · 2016Review
- Evaluation of New Fluorescent Lipophosphoramidates for Gene Transfer and Biodistribution Studies after Systemic Administration.International journal of molecular sciences · 2015Article
- Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy.Proceedings of the National Academy of Sciences of the United States of America · 2015Article
- Differential bioreactivity of neutral, cationic and anionic polystyrene nanoparticles with cells from the human alveolar compartment: robust response of alveolar type 1 epithelial cells.Particle and fibre toxicology · 2015Article
Corrections and comments
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Authors and funding
29 authors at 6 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We use both large and small animal models in our pre-clinical evaluation of gene transfer agents (GTAs) for cystic fibrosis (CF) gene therapy. Here, we report the use of a large animal model to assess three non-viral GTAs: 25 kDa-branched polyethyleneimine (PEI), the cationic liposome (GL67A) and compacted DNA nanoparticle formulated with polyethylene glycol-substituted lysine 30-mer. GTAs complexed with plasmids expressing human cystic fibrosis transmembrane conductance regulator (CFTR) complementary DNA were administered to the sheep lung (n=8 per group) by aerosol. All GTAs gave evidence of gene transfer and expression 1 day after treatment. Vector-derived mRNA was expressed in lung tissues, including epithelial cell-enriched bronchial brushing samples, with median group values reaching 1-10% of endogenous CFTR mRNA levels. GL67A gave the highest levels of expression. Human CFTR protein was detected in small airway epithelial cells in some animals treated with GL67A (two out of eight) and PEI (one out of eight). Bronchoalveolar lavage neutrophilia, lung histology and elevated serum haptoglobin levels indicated that gene delivery was associated with mild local and systemic inflammation. Our conclusion was that GL67A was the best non-viral GTA currently available for aerosol delivery to the sheep lung, led to the selection of GL67A as our lead GTA for clinical trials in CF patients.
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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.