ArticleNeuron2025
Specific targeting of brain endothelial cells using enhancer AAV vectors.
Article in Neuron, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
What it found
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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.
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Who cites it
8 citing papers in PubMed.
- Article
- Neuropixels Opto: combining high-resolution electrophysiology and optogenetics.Nature methods · 2026Article
- Towards precision medicine for brain arteriovenous malformations.The Journal of clinical investigation · 2026Review
- Mitochondrial-Endothelial Crosstalk in Cardiometabolic Disease: Mechanisms and Translational Opportunities in the Multi-omics Era.Reviews in cardiovascular medicine · 2026Review
- Defining a Midgestational Window forbioRxiv : the preprint server for biology · 2026Article
- Endothelial miR-7052 Safeguards Blood-Brain Barrier Integrity During Endotoxemia by Co-repressing ANGPT2 and PDE5A.Molecular neurobiology · 2026Article
- Brain Endothelial Glycocalyx as a Blood-Facing Translational Interface in Alzheimer's Disease: Beyond "Leaky" Barriers Toward Repair-First Stratification.Drug design, development and therapy · 2026Review
- Combining Machine Learning and Multiplexed,bioRxiv : the preprint server for biology · 2025Article
Corrections and comments
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Authors and funding
14 authors.
Funding
Abstract
Brain endothelial cells (BECs) in brain vasculature are critical structural and functional components of the blood brain barrier (BBB). Adeno-associated virus (AAV) capsids have previously been genetically engineered to confer specificity to endothelial cells, but these capsids show limited endothelial cell specificity that varies by delivery conditions. We developed a set of new BEC-enhancer AAV vectors that specifically target BECs based on the cis-regulatory elements identified from single-cell epigenetic datasets. Ex vivo and in vivo characterization of BEC-enhancer AAVs in wild-type, Ai9 reporter, and Alzheimer's disease model mouse brains show their utility for high transduction selectivity of the BECs with little off-target transduction in the liver. Our BEC-enhancer AAVs target the brain vasculature by systemic administration and can be minimally invasive in terms of the route of administration. They are useful new tools for delivering genetic payloads specifically to BECs for normal and diseased brain studies.
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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.