ArticlePloS one2014
Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression.
Article in PloS one, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers.
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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.
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Who cites it
37 citing papers in PubMed, 53 citations in OpenAlex.
- Whole-brain biodistribution of AAV2, AAV8, and AAV-B10 after unilateral intracerebroventricular injection.Molecular therapy. Advances · 2026Article
- CD8+ T cell activation occurs 24 hours after AAV administration and is driven by muscle promoter specificity.JCI insight · 2026Article
- Growth differentiation factor 10 inhibits fat infiltration in tongue muscles of mice with high-fat diet.Skeletal muscle · 2025Article
- rAAV expressing a COBRA-designed influenza hemagglutinin generates a protective and durable adaptive immune response with a single dose.Journal of virology · 2024Article
- Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration.Nature biotechnology · 2024Article
- Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges.Genes & diseases · 2024Review
- Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid.Journal of virology · 2023Article
- Biodegradable Polyester Nanoparticle Vaccines Deliver Self-Amplifying mRNA in Mice at Low Doses.Advanced therapeutics · 2023Article
- Engineered AAV8 capsid acquires heparin and AVB sepharose binding capacity but has altered in vivo transduction efficiency.Gene therapy · 2023Article
- Adeno-associated virus-vectored delivery of HIV biologics: the promise of a "single-shot" functional cure for HIV infection.Journal of virus eradication · 2023Article
- rAAV expressing recombinant antibody for emergency prevention and long-term prophylaxis of COVID-19.Frontiers in immunology · 2023Article
- A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors.Frontiers in immunology · 2023Article
- Single-dose AAV-based vaccine induces a high level of neutralizing antibodies against SARS-CoV-2 in rhesus macaques.Protein & cell · 2023Article
- AAV- based vector improvements unrelated to capsid protein modification.Frontiers in medicine · 2023Review
- Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases.Human gene therapy · 2022Article
- rAAV expressing recombinant neutralizing antibody for the botulinum neurotoxin type A prophylaxis.Frontiers in microbiology · 2022Article
- Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction.Frontiers in immunology · 2021Article
- High-Resolution Histological Landscape of AAV DNA Distribution in Cellular Compartments and Tissues following Local and Systemic Injection.Molecular therapy. Methods & clinical development · 2020Article
- Intralingual Administration of AAVrh10-miRHuman gene therapy · 2020Article
- Liver-Directed but Not Muscle-Directed AAV-Antibody Gene Transfer Limits Humoral Immune Responses in Rhesus Monkeys.Molecular therapy. Methods & clinical development · 2020Article
Corrections and comments
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Authors and funding
12 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Intramuscular (IM) administration of adeno-associated viral (AAV) vectors has entered the early stages of clinical development with some success, including the first approved gene therapy product in the West called Glybera. In preparation for broader clinical development of IM AAV vector gene therapy, we conducted detailed pre-clinical studies in mice and macaques evaluating aspects of delivery that could affect performance. We found that following IM administration of AAV8 vectors in mice, a portion of the vector reached the liver and hepatic gene expression contributed significantly to total expression of secreted transgenes. The contribution from liver could be controlled by altering injection volume and by the use of traditional (promoter) and non-traditional (tissue-specific microRNA target sites) expression control elements. Hepatic distribution of vector following IM injection was also noted in rhesus macaques. These pre-clinical data on AAV delivery should inform safe and efficient development of future AAV products.
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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.