ArticleGene therapy2003
Selective gene expression in brain microglia mediated via adeno-associated virus type 2 and type 5 vectors.
Article in Gene therapy, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 56 papers.
What it found
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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.
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
56 citing papers in PubMed, 101 citations in OpenAlex.
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- Review
- Simple and highly specific targeting of resident microglia with adeno-associated virus.iScience · 2024Article
- A comprehensive review of AAV-mediated strategies targeting microglia for therapeutic intervention of neurodegenerative diseases.Journal of neuroinflammation · 2024Review
- Use of adeno-associated viruses for transgenic modulation of microglia structure and function: A review of technical considerations and challenges.Brain, behavior, and immunity · 2024Review
- Simple and Highly Specific Targeting of Resident Microglia with Adeno-Associated Virus.bioRxiv : the preprint server for biology · 2023Article
- Gene and Cellular Therapies for Leukodystrophies.Pharmaceutics · 2023Review
- Targeted evolution of adeno-associated virus capsids for systemic transgene delivery to microglia and tissue-resident macrophages.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- Microglia dynamics in aging-related neurobehavioral and neuroinflammatory diseases.Journal of neuroinflammation · 2022Review
- Development of microglia-targeting adeno-associated viral vectors as tools to study microglial behavior in vivo.Communications biology · 2022Article
- Directed evolution of adeno-associated virus for efficient gene delivery to microglia.Nature methods · 2022Article
- The use of viral vectors to promote repair after spinal cord injury.Experimental neurology · 2022Review
- Microglia-Specific Promoter Activities ofFrontiers in cellular neuroscience · 2022Article
- Optimizing AAV2/6 microglial targeting identified enhanced efficiency in the photoreceptor degenerative environment.Molecular therapy. Methods & clinical development · 2021Article
- M2 macrophages, but not M1 macrophages, support megakaryopoiesis by upregulating PI3K-AKT pathway activity.Signal transduction and targeted therapy · 2021Article
- Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters.Gene therapy · 2021Article
- Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System.Frontiers in genome editing · 2021Review
- Review
- Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.Nucleic acids research · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
Microglia represent a crucial cell population in the central nervous system, participating in the regulation and surveillance of physiological processes as well as playing key roles in the etiologies of several major brain disorders. The ability to target gene transfer vehicles selectively to microglia would provide a powerful new approach to investigations of mechanisms regulating brain pathologies, as well as enable the development of novel therapeutic strategies. In this study, we evaluate the feasibility of specifically and efficiently targeting microglia relative to other brain cells, using vectors based on two different serotypes of adeno-associated virus (AAV) carrying cell-type-specific transcriptional elements to regulate gene expression. Among a set of promoter choices examined, an element derived from the gene for the murine macrophage marker F4/80 was the most discriminating for microglia. Gene expression from vectors controlled by this element was highly selective for microglia, both in vitro and in vivo. To our knowledge, this is the first demonstration of selective expression of transferred genes in microglia using AAV-derived vectors, as well as the first utilization of recombinant AAV-5 vectors in any macrophage lineage. These results provide strong encouragement for the application of these vectors and this approach for delivering therapeutic and other genes selectively to microglia.
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What OpenQuestion holds
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.