ReviewThe Biochemical journal2005
Novel molecular approaches to cystic fibrosis gene therapy.
Review in The Biochemical journal, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
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, 1 synthesis or guideline pooled it, 90 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
- Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.Gene therapy · 2019Review
- Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer.Journal of cardiovascular development and disease · 2019Review
- A European regulatory perspective on cystic fibrosis: current treatments, trends in drug development and translational challenges for CFTR modulators.European respiratory review : an official journal of the European Respiratory Society · 2018Review
- Pseudomonas aeruginosa and Periodontal Pathogens in the Oral Cavity and Lungs of Cystic Fibrosis Patients: a Case-Control Study.Journal of clinical microbiology · 2015Article
- Extracellular vesicles and their synthetic analogues in aging and age-associated brain diseases.Biogerontology · 2015Review
- In vivo evaluation of adeno-associated virus gene transfer in airways of mice with acute or chronic respiratory infection.Human gene therapy · 2014Article
- CFTR Modulators for the Treatment of Cystic Fibrosis.P & T : a peer-reviewed journal for formulary management · 2014Article
- RNA interference with special reference to combating viruses of crustacea.Indian journal of virology : an official organ of Indian Virological Society · 2012Article
- A synthetic chloride channel restores chloride conductance in human cystic fibrosis epithelial cells.PloS one · 2012Article
- Augmentation of adenovirus 5 vector-mediated gene transduction under physiological pH conditions by a chitosan/NaHCO3 solution.Gene therapy · 2011Article
- Improvement of different vaccine delivery systems for cancer therapy.Molecular cancer · 2011Review
- Nanodelivery in airway diseases: challenges and therapeutic applications.Nanomedicine : nanotechnology, biology, and medicine · 2010Review
- Lentiviral vectors and cystic fibrosis gene therapy.Viruses · 2010Article
- Optimization of Streptomyces bacteriophage phi C31 integrase system to prevent post integrative gene silencing in pulmonary type II cells.Experimental & molecular medicine · 2009Article
- DNA immunization as an efficient strategy for vaccination.Avicenna journal of medical biotechnology · 2009Article
- Gene therapy approaches for stem cell protection.Gene therapy · 2008Review
- Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung.Proceedings of the National Academy of Sciences of the United States of America · 2005Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy holds promise for the treatment of a range of inherited diseases, such as cystic fibrosis. However, efficient delivery and expression of the therapeutic transgene at levels sufficient to result in phenotypic correction of cystic fibrosis pulmonary disease has proved elusive. There are many reasons for this lack of progress, both macroscopically in terms of airway defence mechanisms and at the molecular level with regard to effective cDNA delivery. This review of approaches to cystic fibrosis gene therapy covers these areas in detail and highlights recent progress in the field. For gene therapy to be effective in patients with cystic fibrosis, the cDNA encoding the cystic fibrosis transmembrane conductance regulator protein must be delivered effectively to the nucleus of the epithelial cells lining the bronchial tree within the lungs. Expression of the transgene must be maintained at adequate levels for the lifetime of the patient, either by repeat dosage of the vector or by targeting airway stem cells. Clinical trials of gene therapy for cystic fibrosis have demonstrated proof of principle, but gene expression has been limited to 30 days at best. Results suggest that viral vectors such as adenovirus and adeno-associated virus are unsuited to repeat dosing, as the immune response reduces the effectiveness of each subsequent dose. Nonviral approaches, such as cationic liposomes, appear more suited to repeat dosing, but have been less effective. Current work regarding non-viral gene delivery is now focused on understanding the mechanisms involved in cell entry, endosomal escape and nuclear import of the transgene. There is now increasing evidence to suggest that additional ligands that facilitate endosomal escape or contain a nuclear localization signal may enhance liposome-mediated gene delivery. Much progress in this area has been informed by advances in our understanding of the mechanisms by which viruses deliver their genomes to the nuclei of host cells.
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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.