ArticleMolecular therapy. Methods & clinical development2018
An Adeno-Associated Viral Vector Capable of Penetrating the Mucus Barrier to Inhaled Gene Therapy.
Article in Molecular therapy. Methods & clinical development, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 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
38 citing papers in PubMed, 63 citations in OpenAlex.
- Treatment of pulmonary fibrosis: From disease mechanisms to future novel therapies (Review).International journal of molecular medicine · 2026Review
- Barrier Function of the Extracellular Matrix in AAV Gene Therapy.Cellular and molecular bioengineering · 2026Article
- Article
- A highly mobile adeno-associated virus targeting vascular smooth muscle cells for the treatment of pulmonary arterial hypertension.Nature biomedical engineering · 2025Article
- Simulation of drug transport in airway surface liquid considering mucus flow and ciliary interaction.Scientific reports · 2025Article
- An adeno-associated virus gene therapy strategy for anti-obesity treatment by nanocarrier-based delivery systems.Journal of nanobiotechnology · 2025Article
- Review
- Recent Advances in Therapeutics and Manufacturing Processes of Recombinant Adeno-Associated Virus for the Treatment of Lung Diseases.Current gene therapy · 2025Review
- Recent Developments in Aerosol Pulmonary Drug Delivery: New Technologies, New Cargos, and New Targets.Annual review of biomedical engineering · 2024Review
- Synthetic mucus barrier arrays as a nanoparticle formulation screening platform.RSC pharmaceutics · 2024Article
- Recent Progress in Nucleic Acid Pulmonary Delivery toward Overcoming Physiological Barriers and Improving Transfection Efficiency.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Review
- Surface Cross-Linking by Macromolecular Tethers Enhances Virus-like Particles' Resilience to Mucosal Stress Factors.ACS nano · 2024Article
- Inhalable Bottlebrush Polymer Bioconjugates as Vectors for Efficient Pulmonary Delivery of Oligonucleotides.ACS nano · 2024Article
- Airway basal cells from human-induced pluripotent stem cells: a new frontier in cystic fibrosis research.Frontiers in cell and developmental biology · 2024Article
- Multiple Particle Tracking: A Method for Probing Biologically Relevant Mobility of Bacterial Extracellular Vesicles.Methods in molecular biology (Clifton, N.J.) · 2024Article
- Synthetic mucus barrier arrays as a nanoparticle formulation screening platform.bioRxiv : the preprint server for biology · 2023Article
- Computational fluid dynamics modeling of aerosol particle transport through lung airway mucosa.Computers & chemical engineering · 2023Article
- 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
- Inhaled drug delivery for the targeted treatment of asthma.Advanced drug delivery reviews · 2023Review
- Article
Corrections and comments
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Authors and funding
13 authors at 7 institutions in 1 country.
Funding
Abstract
Diffusion of the viral vectors evaluated in inhaled gene therapy clinical trials to date are largely hindered within airway mucus, which limits their access to, and transduction of, the underlying airway epithelium prior to clearance from the lung. Here, we discovered that adeno-associated virus (AAV) serotype 6 was able to rapidly diffuse through mucus collected from cystic fibrosis (CF) patients, unlike previously tested AAV serotypes. A point mutation of the AAV6 capsid suggests a potential mechanism by which AAV6 avoids adhesion to the mucus mesh. Significantly greater transgene expression was achieved with AAV6 compared to a mucoadhesive serotype, AAV1, in air-liquid interface cultures of human CF bronchial epithelium with naturally secreted mucus or induced mucus hypersecretion. In addition, AAV6 achieved superior distribution and overall level of transgene expression compared to AAV1 in the airways and whole lungs, respectively, of transgenic mice with airway mucus obstruction. Our findings motivate further evaluation and clinical development of AAV6 for inhaled 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.