Targeted gene-delivery tools for brain immune cells (microglia)
Directed Evolution of Novel AAVs and Regulatory Elements for Selective Microglial Gene Expression
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA BERKELEY · NIH-11323566
This project will create viral gene-delivery tools that specifically target microglia in adult primate and human brain tissue to enable future therapies for Alzheimer's and other brain disorders.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA BERKELEY (nih funded) |
| Locations | 1 site (BERKELEY, UNITED STATES) |
| Trial ID | NIH-11323566 on ClinicalTrials.gov |
What this research studies
Researchers will engineer many versions of harmless AAV viral vectors and use directed evolution to find versions that enter microglia in adult primate and human tissue. They will add molecular barcodes to track which viral variants reach microglia, use single-cell sequencing and machine learning to read results, and test regulatory DNA elements to make gene expression specific to microglia. Experiments will use human brain tissue and non-human primate models rather than testing new therapies in patients at this stage. The goal is to build a platform that reliably and selectively delivers genes to microglia so future treatments can target harmful inflammation without affecting neurons.
Who could benefit from this research
Good fit: Adults with Alzheimer's disease, Parkinson's disease, ALS, Huntington's disease, certain neurodevelopmental disorders, or neuropathic pain linked to microglial inflammation could be future candidates for therapies developed from this work.
Not a fit: People with non-neurological conditions or those needing immediate, currently approved treatments are unlikely to receive direct benefit from this preclinical project.
Why it matters
Potential benefit: If successful, this could enable gene therapies that reduce harmful microglial inflammation and slow or prevent progression of Alzheimer's and other neurodegenerative or neurodevelopmental conditions.
How similar studies have performed: Viral engineering and directed evolution have produced AAVs that target neurons and other brain cells, but reliably and selectively targeting microglia in adult primate brains is largely novel and unproven.
Where this research is happening
BERKELEY, UNITED STATES
- UNIVERSITY OF CALIFORNIA BERKELEY — BERKELEY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SCHAFFER, DAVID V — UNIVERSITY OF CALIFORNIA BERKELEY
- Study coordinator: SCHAFFER, DAVID V
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: Alzheimer disease dementia, Alzheimer syndrome