ArticleProceedings of the National Academy of Sciences of the United States of America2003
Protection of Cftr knockout mice from acute lung infection by a helper-dependent adenoviral vector expressing Cftr in airway epithelia.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
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
33 citing papers in PubMed, 79 citations in OpenAlex.
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- The Potential Relevance of PnDREBs to Panax notoginseng Nitrogen Sensitiveness.Biochemical genetics · 2024Article
- Effective viral-mediated lung gene therapy: is airway surface preparation necessary?Gene therapy · 2023Review
- Gene therapy for cystic fibrosis: Challenges and prospects.Frontiers in pharmacology · 2022Review
- Gene therapy for cystic fibrosis: new tools for precision medicine.Journal of translational medicine · 2021Review
- Article
- Overcoming the Undesirable CRISPR-Cas9 Expression in Gene Correction.Molecular therapy. Nucleic acids · 2018Article
- Review
- Review
- Cystic Fibrosis Gene Therapy in the UK and Elsewhere.Human gene therapy · 2015Review
- Testing gene therapy vectors in human primary nasal epithelial cultures.Molecular therapy. Methods & clinical development · 2015Article
- Transient blocking of NK cell function with small molecule inhibitors for helper dependant adenoviral vector-mediated gene delivery.Cell & bioscience · 2015Article
- Lung gene therapy-How to capture illumination from the light already present in the tunnel.Genes & diseases · 2014Article
- Efficient gene delivery to pig airway epithelia and submucosal glands using helper-dependent adenoviral vectors.Molecular therapy. Nucleic acids · 2013Article
- Gene therapy: light is finally in the tunnel.Protein & cell · 2011Review
- Helper-Dependent Adenoviral Vectors.Journal of genetic syndromes & gene therapy · 2011Article
- Chemopreventive efficacy and mechanism of licofelone in a mouse lung tumor model via aspiration.Cancer prevention research (Philadelphia, Pa.) · 2011Article
- Subretinal gene delivery using helper-dependent adenoviral vectors.Cell & bioscience · 2011Article
- Readministration of helper-dependent adenoviral vectors to mouse airway mediated via transient immunosuppression.Gene therapy · 2011Article
- Gene therapy with helper-dependent adenoviral vectors: current advances and future perspectives.Viruses · 2010Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 4 institutions in 1 country.
Funding
Abstract
We developed a helper-dependent adenoviral vector for cystic fibrosis lung gene therapy. The vector expresses cystic fibrosis transmembrane conductance regulator (Cftr) using control elements from cytokeratin 18. The vector expressed properly localized CFTR in cultured cells and in the airway epithelia of mice. Cftr RNA and protein were present in whole lung and bronchioles, respectively, for 28 days after a vector dose. Acute inflammation was minimal to moderate. To test the therapeutic potential of the vector, we challenged mice with a clinical strain of Burkholderia cepacia complex (Bcc). Cftr knockout mice (but not Cftr+/+ littermates) challenged with Bcc developed severe lung histopathology and had high lung bacteria counts. Cftr knockout mice receiving gene therapy 7 days before Bcc challenge had less severe histopathology, and the number of lung bacteria was reduced to the level seen in Cftr+/+ littermates. These data suggest that gene therapy could benefit cystic fibrosis patients by reducing susceptibility to opportunistic pathogens.
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.