ReviewAdvanced drug delivery reviews2002
Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis.
Review in Advanced drug delivery reviews, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 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
51 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
- Lipid nanoparticles optimized for large RNA cargo and tissue targeting enhance in vivo genome editing.Nature biotechnology · 2026Article
- Extracellular vesicle-based delivery to airway basal cells for durable gene therapy in cystic fibrosis.Frontiers in bioengineering and biotechnology · 2026Review
- High ionic strength vector formulations enhance gene transfer to airway epithelia.Nucleic acids research · 2024Article
- Article
- Reduced miR-513a-5p expression in COPD may regulate airway mucous cell hyperplasia through TFR1-dependent signaling.The Kaohsiung journal of medical sciences · 2024Article
- Progress in Respiratory Gene Therapy.Human gene therapy · 2022Review
- A Two-Pronged Pulmonary Gene Delivery Strategy: A Surface-Modified Fullerene Nanoparticle and a Hypotonic Vehicle.Angewandte Chemie (International ed. in English) · 2021Article
- Single-Dose Lentiviral Mediated Gene Therapy Recovers CFTR Function in Cystic Fibrosis Knockout Rats.Frontiers in pharmacology · 2021Article
- Physiological study of pulmonary involvement in adults with cystic fibrosis through simulated modeling of different clinical scenarios.Medical & biological engineering & computing · 2019Article
- An Adeno-Associated Viral Vector Capable of Penetrating the Mucus Barrier to Inhaled Gene Therapy.Molecular therapy. Methods & clinical development · 2018Article
- The Mucus Barrier to Inhaled Gene Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- Article
- CRISPR-Cas9 directed knock-out of a constitutively expressed gene using lance array nanoinjection.SpringerPlus · 2016Article
- Cell Penetrating Peptide Conjugated Chitosan for Enhanced Delivery of Nucleic Acid.International journal of molecular sciences · 2015Review
- New and Emerging Treatments for Cystic Fibrosis.Drugs · 2015Review
- In vivo evaluation of adeno-associated virus gene transfer in airways of mice with acute or chronic respiratory infection.Human gene therapy · 2014Article
- Nanotechnology approaches for inhalation treatment of fibrosis.Journal of drug targeting · 2013Review
- Efficient delivery of RNA interference oligonucleotides to polarized airway epithelia in vitro.American journal of physiology. Lung cellular and molecular physiology · 2013Article
- Dual-degradable disulfide-containing PEI-Pluronic/DNA polyplexes: transfection efficiency and balancing protection and DNA release.International journal of nanomedicine · 2013Article
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
Abstract
Clinical trials of gene therapy for cystic fibrosis suggest that current levels of gene transfer efficiency are probably too low to result in clinical benefit, largely as a result of the barriers faced by gene transfer vectors within the airways. The respiratory epithelium has evolved a complex series of extracellular barriers (mucus, lack of receptors, immune surveillance, etc.) aimed at preventing penetration of lumenally delivered materials, including gene therapy vectors. In addition, once in the cell, further hurdles have to be overcome, including DNA degradation, nuclear import and the ability to maintain long-term transgene expression. Strategies to overcome these barriers will be addressed in this review and include the use of: (i) clinically relevant adjuncts to overcome the extra- and intracellular barriers; (ii) less-conventional delivery routes, such as intravenous or in utero administration; (iii) more efficient non-viral vectors and 'stealth' viruses which can be re-administered; and (iv) new approaches to prolong transgene expression by means of alternative promoters or integrating vectors. These advances have the potential to improve the efficiency of gene delivery to the airway epithelium, thus making gene therapy a more realistic option for cystic fibrosis.
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.