Trial reportNature medicine1995
Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.
Trial report in Nature medicine, 1995. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 141 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
141 citing papers in PubMed, 707 citations in OpenAlex.
- Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis.Gene therapy · 2000Trial
- Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus.The Journal of clinical investigation · 1999Trial
- Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo.The Journal of clinical investigation · 1997Trial
- The role of CFTR in spermatogenesis and sperm quality.Nature reviews. Urology · 2026Review
- Genetically engineered approaches to the treatment of cystic fibrosis.Biophysical reviews · 2025Review
- 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
- Non-Viral Delivery Systems to Transport Nucleic Acids for Inherited Retinal Disorders.Pharmaceuticals (Basel, Switzerland) · 2025Review
- A case study of the informative value of risk of bias and reporting quality assessments for systematic reviews.Systematic reviews · 2024Article
- Potential therapeutic strategies for osteoarthritis via CRISPR/Cas9 mediated gene editing.Reviews in endocrine & metabolic disorders · 2024Review
- Molecular and functional correction of a deep intronic splicing mutation inMolecular therapy. Methods & clinical development · 2023Article
- 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
- Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Lipid-based nucleic acid therapeutics with in vivo efficacy.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology · 2023Review
- Gene therapy for cystic fibrosis: new tools for precision medicine.Journal of translational medicine · 2021Review
- Gene Therapy: A Possible Alternative to CFTR Modulators?Frontiers in pharmacology · 2021Article
- Small interfering RNA for cancer treatment: overcoming hurdles in delivery.Acta pharmaceutica Sinica. B · 2020Review
- Engineered mutant α-ENaC subunit mRNA delivered by lipid nanoparticles reduces amiloride currents in cystic fibrosis-based cell and mice models.Science advances · 2020Article
- T-cell targeted pulmonary siRNA delivery for the treatment of asthma.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology · 2020Review
- Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing.International journal of molecular sciences · 2020Review
81 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
- Commented on by
- Erratum issued
Authors and funding
10 authors at 4 institutions in 2 countries.
Funding
Abstract
We report the results of a double-blind, placebo-controlled trial in nine cystic fibrosis (CF) subjects receiving cationic liposome complexed with a complementary DNA encoding the CF transmembrane conductance regulator (CFTR), and six CF subjects receiving only liposome to the nasal epithelium. No adverse clinical effects were seen and nasal biopsies showed no histological or immuno-histological changes. A partial restoration of the deficit between CF and non-CF subjects of 20% was seen for the response to low Cl- perfusion following CFTR cDNA administration. This was maximal around day three and had reverted to pretreatment values by day seven. In some cases the response to low Cl- was within the range for non-CF subjects. Plasmid DNA and transgene-derived RNA were detected in the majority of treated subjects. Although these data are encouraging, it is likely that transfection efficiency and the duration of expression will need to be increased for therapeutic benefit.
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.