ArticleThe application of clinical genetics2012
Gene therapy for the treatment of cystic fibrosis.
Article in The application of clinical genetics, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 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
18 citing papers in PubMed, 48 citations in OpenAlex.
- Optimization of chemical transfection in airway epithelial cell lines.BMC biotechnology · 2025Article
- Review
- Respiratory Infection and Inflammation in Cystic Fibrosis: A Dynamic Interplay among the Host, Microbes, and Environment for the Ages.International journal of molecular sciences · 2023Review
- Progress in Respiratory Gene Therapy.Human gene therapy · 2022Review
- Chemically modified mRNA beyond COVID-19: Potential preventive and therapeutic applications for targeting chronic diseases.Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · 2022Review
- Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.Molecular neurobiology · 2022Review
- CRISPR Gene-Editing Models Geared Toward Therapy for Hereditary and Developmental Neurological Disorders.Frontiers in pediatrics · 2021Review
- New Therapeutic Approaches in Cystic Fibrosis.Turkish journal of pharmaceutical sciences · 2020Article
- Gene Therapy for Respiratory Diseases: Progress and a Changing Context.Human gene therapy · 2020Article
- Article
- Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity.JAMA network open · 2020Article
- Novel therapeutic approaches for the management of cystic fibrosis.Multidisciplinary respiratory medicine · 2020Article
- Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.Gene therapy · 2019Review
- Transcriptome Profiling and Molecular Therapeutic Advances in Cystic Fibrosis: Recent Insights.Genes · 2019Review
- Adeno-Associated Virus (AAV) as a Vector for Gene Therapy.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2017Review
- Molecular Imaging in Genetic Medicine.Cureus · 2016Review
- Unmet needs in respiratory diseases : "You can't know where you are going until you know where you have been"--Anonymous.Clinical reviews in allergy & immunology · 2013Review
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has hitherto been widely-researched yet for which no treatment exists that halts the progression of lung disease. Gene therapy involves the transfer of correct copies of cystic fibrosis transmembrane conductance regulator (CFTR) DNA to the epithelial cells in the airways. The cloning of the CFTR gene in 1989 led to proof-of-principle studies of CFTR gene transfer in vitro and in animal models. The earliest clinical trials in CF patients were conducted in 1993 and used viral and non-viral gene transfer agents in both the nasal and bronchial airway epithelium. To date, studies have focused largely on molecular or bioelectric (chloride secretion) outcome measures, many demonstrating evidence of CFTR expression, but few have attempted to achieve clinical efficacy. As CF is a lifelong disease, turnover of the airway epithelium necessitates repeat administration. To date, this has been difficult to achieve with viral gene transfer agents due to host recognition leading to loss of expression. The UK Cystic Fibrosis Gene Therapy Consortium (Imperial College London, University of Edinburgh and University of Oxford) is currently working on a large and ambitious program to establish the clinical benefits of CF gene therapy. Wave 1, which has reached the clinic, uses a non-viral vector. A single-dose safety trial is nearing completion and a multi-dose clinical trial is shortly due to start; this will be powered for clinically-relevant changes. Wave 2, more futuristically, will look at the potential of lentiviruses, which have long-lasting expression. This review will summarize the current status of translational research in CF gene therapy.
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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.