ReviewGene therapy2004
Gene therapy for cystic fibrosis: an example for lung gene therapy.
Review in Gene therapy, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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
15 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease.The Cochrane database of systematic reviews · 2016Pooled it
- Tyrosine-Mutant AAV8 Vector Mediated Efficient and Safe Gene Transfer of Pigment Epithelium-Derived Factor to Mouse Lungs.Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology · 2023Article
- Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid.Molecular therapy. Methods & clinical development · 2022Article
- T-cell targeted pulmonary siRNA delivery for the treatment of asthma.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology · 2020Review
- Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis.Scientific reports · 2018Article
- Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique.Frontiers in pharmacology · 2018Review
- Drug discovery and development for rare genetic disorders.American journal of medical genetics. Part A · 2017Review
- Article
- Regulation of Pulmonary Vascular Smooth Muscle Contractility in Pulmonary Arterial Hypertension: Implications for Therapy.Frontiers in physiology · 2017Review
- Advances in cell and gene-based therapies for cystic fibrosis lung disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Review
- Human amnion epithelial cells induced to express functional cystic fibrosis transmembrane conductance regulator.PloS one · 2012Article
- Common gene therapy viral vectors do not efficiently penetrate sputum from cystic fibrosis patients.PloS one · 2011Article
- Gene therapy for children with cystic fibrosis--who has the right to choose?Journal of medical ethics · 2006Review
- Canine adenovirus vectors for lung-directed gene transfer: efficacy, immune response, and duration of transgene expression using helper-dependent vectors.Journal of virology · 2006Article
- Mechanism of efficient transfection of the nasal airway epithelium by hypotonic shock.Gene therapy · 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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy is currently being evaluated for a wide range of acute and chronic lung diseases. The requirement of gene transfer into the individual cell types of the complex lung structure will very much depend on the target disease. Over the last decade, the gene therapy community has recognized that there is not even one vector that is good for all applications, but that the gene transfer agent has to be carefully chosen. Gene therapy is particularly attractive for diseases that currently do not have satisfactory treatment options and probably easier for monogenic disorders than for complex diseases. Cystic fibrosis (CF) fulfills these criteria and is therefore a good candidate for gene therapy-based treatment. This review will focus on CF as an example for lung gene therapy and discuss the progress made in this field over the last couple of years.
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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.