Trial reportThe Journal of clinical investigation1997
Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo.
Trial report in The Journal of clinical investigation, 1997. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
36 citing papers in PubMed, 1 synthesis or guideline pooled it, 177 citations in OpenAlex.
- Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease.The Cochrane database of systematic reviews · 2016Pooled it
- 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
- Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis.Gene therapy · 2000Trial
- The Era of Gene Therapy: The Advancement of Lentiviral Vectors and Their Pseudotyping.Viruses · 2025Review
- Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy.DNA and cell biology reports · 2025Article
- The arc of discovery, from the description of cystic fibrosis to effective treatments.The Journal of clinical investigation · 2024Article
- 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
- Aerosolizable Plasmid DNA Dry Powders Engineered by Thin-film Freezing.Pharmaceutical research · 2023Article
- Lipid-based nucleic acid therapeutics with in vivo efficacy.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology · 2023Review
- Gene Therapy: A Possible Alternative to CFTR Modulators?Frontiers in pharmacology · 2021Article
- Review
- Review
- Nonviral Gene Therapy for Cancer: A Review.Diseases (Basel, Switzerland) · 2018Review
- Cystic Fibrosis Gene Therapy in the UK and Elsewhere.Human gene therapy · 2015Review
- Lung gene therapy-How to capture illumination from the light already present in the tunnel.Genes & diseases · 2014Article
- Rapid identification of novel functional promoters for gene therapy.Journal of molecular medicine (Berlin, Germany) · 2012Article
- Advances in cell and gene-based therapies for cystic fibrosis lung disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Review
- Gene therapy for the treatment of cystic fibrosis.The application of clinical genetics · 2012Article
- Gene therapy: light is finally in the tunnel.Protein & cell · 2011Review
- Nanodelivery in airway diseases: challenges and therapeutic applications.Nanomedicine : nanotechnology, biology, and medicine · 2010Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cationic lipids show promise as vectors for transfer of CFTR cDNA to airway epithelia of patients with cystic fibrosis (CF). However, previous studies have not compared the effect of DNA-lipid to DNA alone. Recently, we developed a formulation of plasmid encoding CFTR (pCF1-CFTR) and cationic lipid (GL-67:DOPE) that generated greater gene transfer in mouse lung than previously described DNA-lipid vectors. Therefore, we tested the hypothesis that DNA-lipid complexes were more effective than DNA alone at transferring CFTR cDNA to airway epithelia in vivo. We administered complexes of DNA-lipid to one nostril and DNA alone to the other nostril in a randomized, double-blind study. Electrophysiologic measurements showed that DNA-lipid complexes partially corrected the Cl- transport defect. Importantly, the pCF1-CFTR plasmid alone was at least as effective as complexes of DNA with lipid. Measurements of vector-specific CFTR transcripts also showed gene transfer with both DNA-lipid and DNA alone. These results indicate that nonviral vectors can transfer CFTR cDNA to airway epithelia and at least partially restore the Cl- transport defect characteristic of CF. However, improvements in the overall efficacy of gene transfer are required to develop a treatment for CF.
Indexed as
Identifiers
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.