ArticleMolecular therapy. Methods & clinical development2022
Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid.
Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 22 citations in OpenAlex.
- Discovery of an Antiviral Electron Transfer Process to Create Catalytically Self-Sufficient Viral Restriction Factors.Angewandte Chemie (International ed. in English) · 2026Article
- Recent advancements in improving cross-species applicability of bioengineered AAV capsids.Gene therapy · 2026Review
- Tracheal nebulization of AAV-CRE for lung conditional knockout model construction and whole lung clearing.Biophysics reports · 2026Article
- Intravenous rAAV9 Produces Time-Resolved Parenchymal Labeling Downstream of the Vasculature in Adult Mice.Biology · 2026Article
- Multiscale red blood cell hitchhiking for targeted deep tissue gene delivery in lungs.Nature communications · 2025Article
- Optimal sequencing depth for measuring the concentrations of molecular barcodes.Nucleic acids research · 2025Article
- Advances and Challenges in Adeno-Associated Virus Gene Therapy Applications of Localized Delivery Strategies.Current medical science · 2025Review
- Revolution of AAV in Drug Discovery: From Delivery System to Clinical Application.Journal of medical virology · 2025Review
- Programming tumor evolution with selection gene drives to proactively combat drug resistance.Nature biotechnology · 2025Article
- Recent Advances in Therapeutics and Manufacturing Processes of Recombinant Adeno-Associated Virus for the Treatment of Lung Diseases.Current gene therapy · 2025Review
- Adeno-associated virus-based gene therapy treats inflammatory kidney disease in mice.The Journal of clinical investigation · 2024Article
- Gene therapy with AAV9-SGPL1 in an animal model of lung fibrosis.The Journal of pathology · 2024Article
- Brain-Wide Transgene Expression in Mice by Systemic Injection of Genetically Engineered Exosomes: CAP-Exosomes.Pharmaceuticals (Basel, Switzerland) · 2024Article
- Microglia targeting by adeno-associated viral vectors.Frontiers in immunology · 2024Review
- Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice.Molecular therapy. Methods & clinical development · 2023Article
- Drug delivery systems for CRISPR-based genome editors.Nature reviews. Drug discovery · 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
- Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Intravenous functional gene transfer throughout the brain of non-human primates using AAV.Research square · 2023Article
- Advanced approaches to overcome biological barriers in respiratory and systemic routes of administration for enhanced nucleic acid delivery to the lung.Expert opinion on drug deliveryReview
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Advances in adeno-associated virus (AAV) engineering have provided exciting new tools for research and potential solutions for gene therapy. However, the lung has not received the same tailored engineering as other major targets of debilitating genetic disorders. To address this, here we engineered the surface-exposed residues AA452-458 of AAV9 capsid proteins at the three-fold axis of symmetry and employed a Cre-transgenic-based screening platform to identify AAV capsids targeted to the lung after intravenous delivery in mice. Using a custom image processing pipeline to quantify transgene expression across whole tissue images, we found that one engineered variant, AAV9.452sub.LUNG1, displays dramatically improved transgene expression in lung tissue after systemic delivery in mice. This improved transduction extends to alveolar epithelial type II cells, expanding the toolbox for gene therapy research for diseases specific to the lung.
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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.