ArticleCell reports methods2025
AAV vectors for specific and efficient gene expression in microglia.
Article in Cell reports methods, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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Who cites it
9 citing papers in PubMed.
- Targeting glial PD-1/PD-L1 restores microglial homeostasis and reduces neuronal hyperactivity in an Alzheimer's disease model.Science advances · 2026Article
- A compact GAD67 promoter enables inhibitory neuron-targeted AAV gene therapy for seizure suppression.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Aberrant phase separation from a rare ABI3 mutation drives microglial dysfunction and Alzheimer's risk.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026Article
- Trends in the Engineering of Adeno-Associated Virus (AAV) for Precision Gene Delivery to the Central Nervous System (CNS).International journal of molecular sciences · 2026Review
- Protocol for differentiation and efficient AAV-mediated gene delivery to hiPSC-derived microglia for functional studies.STAR protocols · 2026Article
- Sphingomyelin synthase 1 as a potential upstream amplifier of microglial CSF1R signaling in neuropathic pain.Frontiers in molecular neuroscience · 2026Article
- Partners in plasticity: serotonergic glial interactions in brain circuit remodeling.Frontiers in neuroscience · 2026Review
- Blood-brain barrier-penetrant AAV vectors for cell type-specific gene expression in the mouse brain.Anatomical science international · 2025Review
- KLF7 Blocks MKNK2/HIF-1 Pathway-Mediated M1 Microglia Polarization to Ameliorate Ischemic Stroke-Induced Neurological Injury.Brain and behavior · 2025Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Microglia are crucial targets for therapeutic interventions in diseases like Alzheimer's and stroke, but efficient gene delivery to these immune cells is challenging. We developed an adeno-associated virus (AAV) vector that achieves specific and efficient gene delivery to microglia. This vector incorporates the mIba1 promoter, GFP, miRNA target sequences (miR.Ts), WPRE, and poly(A) signal. Positioning miR.Ts on both sides of WPRE significantly suppressed non-microglial expression, achieving over 90% specificity and more than 60% efficiency in microglia-specific gene expression 3 weeks post-administration. Additionally, this vector enabled GCaMP expression, facilitating real-time calcium dynamics monitoring in microglial processes. Using a blood-brain barrier-penetrant AAV-9P31 capsid variant, intravenous administration resulted in broad and selective microglial GFP expression across the brain. These results establish our AAV vector as a versatile tool for long-term, highly specific, and efficient gene expression in microglia, advancing microglial research and potential therapeutic applications.
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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.