ArticleJournal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism2022
Short regulatory DNA sequences to target brain endothelial cells for gene therapy.
Article in Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed.
- A targeted vector for brain endothelial cell gene delivery and cerebrovascular malformation modelling.Nature biomedical engineering · 2026Article
- Advances in endothelial cell targeting by AAV vectors.Acta biochimica Polonica · 2026Review
- A novel vector design targeting the blood-brain barrier with the brain-specific AAV-BR1 vector enables abluminal protein secretion from brain endothelial cells in vitro.Fluids and barriers of the CNS · 2025Article
- Article
- From bench to bedside: murine models of inherited and sporadic brain arteriovenous malformations.Angiogenesis · 2025Review
- Gene therapy of adeno-associated virus (AAV) vectors in preclinical models of ischemic stroke.CNS neuroscience & therapeutics · 2023Review
- A year in review: brain barriers and brain fluids research in 2022.Fluids and barriers of the CNS · 2023Article
- A novel strategy for delivering Niemann-Pick type C2 proteins across the blood-brain barrier using the brain endothelial-specific AAV-BR1 virus.Journal of neurochemistry · 2023Article
- A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System.Nature cardiovascular research · 2022Article
- Review
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Authors and funding
12 authors.
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Abstract
Gene vectors targeting CNS endothelial cells allow to manipulate the blood-brain barrier and to correct genetic defects in the CNS. Because vectors based on the adeno-associated virus (AAV) have a limited capacity, it is essential that the DNA sequence controlling gene expression is short. In addition, it must be specific for endothelial cells to avoid off-target effects. To develop improved regulatory sequences with selectivity for brain endothelial cells, we tested the transcriptional activity of truncated promoters of eleven (brain) endothelial-specific genes in combination with short regulatory elements, i.e., the woodchuck post-transcriptional regulatory element (W), the CMV enhancer element (C), and a fragment of the first intron of the
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