ArticleiScience2024
New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets.
Article in iScience, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed.
- Targeted AAV gene therapy for neuroblastoma via direct capsid-antibody coupling.EMBO molecular medicine · 2026Article
- Shifting AAV9 tropism by binding moiety insertion enhances transduction of tumor cell lines and human glioblastoma explants.Scientific reports · 2026Article
- Optimized AAV vector enables potent therapeutic rescue of inherited glycosylphosphatidylinositol deficiency in mice.Molecular therapy. Advances · 2026Article
- Metadata assessment of non-human primate studies of AAV9 uncovers potential tissue specific variation in expression efficiency.Gene therapy · 2026Article
- Spatial mapping of barcoded, brain-tropic AAVs using multiplexed RNAMolecular therapy. Advances · 2026Article
- Viral vector-based gene therapies in the clinic: An update.Bioengineering & translational medicine · 2026Review
- Potential new drug sources for the treatment of Parkinson's disease: flavonoid O-glycosides.Molecular biology reports · 2025Review
- Directed evolution of novel AAV variants using the MCMS library for enhanced CNS tropism and reduced liver targeting in mice.Molecular therapy. Methods & clinical development · 2025Article
- A versatile cell line for establishing potency of cell type-specific AAV transgenes.Molecular therapy. Methods & clinical development · 2025Article
- Viral and non-viral cellular therapies for neurodegeneration.Frontiers in medicine · 2025Review
- Review
- Evaluation of BR1 and BI30 AAVs for Brain Endothelial Tropism.ASN neuro · 2024Article
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Although adeno-associated virus 9 (AAV9) has been highly exploited as delivery platform for gene-based therapies, its efficacy is hampered by low efficiency in crossing the adult blood-brain barrier (BBB) and pronounced targeting to the liver upon intravenous delivery. We generated a new galactose binding-deficient AAV9 peptide display library and selected two new AAV9 engineered capsids with enhanced targeting in mouse and marmoset brains after intravenous delivery. Interestingly, the loss of galactose binding greatly reduced undesired targeting to peripheral organs, particularly the liver, while not compromising transduction of the brain vasculature. However, the galactose binding was necessary to efficiently infect non-endothelial brain cells. Thus, the combinatorial actions of the galactose-binding domain and the incorporated displayed peptide are crucial to enhance BBB crossing along with brain cell transduction. This study describes two novel capsids with high brain endothelial infectivity and extremely low liver targeting based on manipulating the AAV9 galactose-binding domain.
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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.