ArticleStem cell research & therapy2020
Identification of AAV serotypes for lung gene therapy in human embryonic stem cell-derived lung organoids.
Article in Stem cell research & therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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
22 citing papers in PubMed, 1 synthesis or guideline pooled it, 32 citations in OpenAlex.
- Pooled it
- Improving the precision of AAV lung gene therapy for SP-B deficiency using computationally derived lung-specific promoters.Gene therapy · 2026Article
- A nanobody-based proteolysis-targeting chimera offers broad-spectrum protection against diverse influenza virus infections.Signal transduction and targeted therapy · 2026Article
- Lung Organoids from hiPSCs Can Be Efficiently Transduced by Recombinant Adeno-Associated Viral and Adenoviral Vectors.Biomedicines · 2025Article
- Optimized gene transduction in human lung organoids: A high-efficiency method for advanced research applications.Communications biology · 2025Article
- Recent Advances in Therapeutics and Manufacturing Processes of Recombinant Adeno-Associated Virus for the Treatment of Lung Diseases.Current gene therapy · 2025Review
- In vitro modelling of bacterial pneumonia: a comparative analysis of widely applied complex cell culture models.FEMS microbiology reviews · 2024Review
- tRNA therapeutics for genetic diseases.Nature reviews. Drug discovery · 2024Review
- Descriptive and Functional Genomics in Neonatal Respiratory Distress Syndrome: From Lung Development to Targeted Therapies.International journal of molecular sciences · 2024Review
- Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice.Molecular therapy. Methods & clinical development · 2023Article
- Repeat or single-dose lentiviral vector administration to mouse lungs? It's all about the timing.Gene therapy · 2023Article
- Advanced models for respiratory disease and drug studies.Medicinal research reviews · 2023Review
- Vectored immunoprophylaxis and treatment of SARS-CoV-2 infection in a preclinical model.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- Advanced lung organoids and lung-on-a-chip for cancer research and drug evaluation: a review.Frontiers in bioengineering and biotechnology · 2023Review
- Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell.Human gene therapy · 2022Review
- Minilungs from Human Embryonic Stem Cells to Study the Interaction of Streptococcus pneumoniae with the Respiratory Tract.Microbiology spectrum · 2022Article
- Organoids and microphysiological systems: Promising models for accelerating AAV gene therapy studies.Frontiers in immunology · 2022Review
- ACE2 Expression in Organotypic Human Airway Epithelial Cultures and Airway Biopsies.Frontiers in pharmacology · 2022Article
- Lentiviral and AAV-mediated expression of palivizumab offer protection against Respiratory Syncytial Virus infection.Scientific reports · 2021Article
- Lung organoids, useful tools for investigating epithelial repair after lung injury.Stem cell research & therapy · 2021Review
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 2 countries.
Funding
Abstract
Gene therapy is being investigated for a range of serious lung diseases, such as cystic fibrosis and emphysema. Recombinant adeno-associated virus (rAAV) is a well-established, safe, viral vector for gene delivery with multiple naturally occurring and artificial serotypes available displaying alternate cell, tissue, and species-specific tropisms. Efficient AAV serotypes for the transduction of the conducting airways have been identified for several species; however, efficient serotypes for human lung parenchyma have not yet been identified. Here, we screened the ability of multiple AAV serotypes to transduce lung bud organoids (LBOs)-a model of human lung parenchyma generated from human embryonic stem cells. Microinjection of LBOs allowed us to model transduction from the luminal surface, similar to dosing via vector inhalation. We identified the naturally occurring rAAV2 and rAAV6 serotypes, along with synthetic rAAV6 variants, as having tropism for the human lung parenchyma. Positive staining of LBOs for surfactant proteins B and C confirmed distal lung identity and suggested the suitability of these vectors for the transduction of alveolar type II cells. Our findings establish LBOs as a new model for pulmonary gene therapy and stress the relevance of LBOs as a viral infection model of the lung parenchyma as relevant in SARS-CoV-2 research.
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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.