Developing targeted gene therapy for brain inflammation in neurological diseases
Official title: Directed Evolution of Novel AAVs and Regulatory Elements for Selective Microglial Gene Expression
Funded at University of California Berkeley · NIH-11031284 · Data updated 2026-11-12
This study is exploring new ways to use gene therapy to help treat brain conditions like Alzheimer's and ALS by targeting specific brain cells, and it's designed for people who are looking for advanced treatments for these diseases.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Berkeley NIH-funded |
| Lab location | 1 site (Berkeley, United States) |
| Project start | March 15, 2023 |
| Project end | February 29, 2028 |
| Funding year | 2025 |
| Award (this year) | $788,079 |
| NIH project number | 5R01NS126397-03 |
| Project ID | NIH-11031284 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with neurodegenerative disorders such as Alzheimer's disease or amyotrophic lateral sclerosis.
✕Less likely to help
Patients with acute neurological injuries or those not diagnosed with the targeted neurodegenerative conditions may not benefit from this research.
What this research studies
This research focuses on creating advanced gene therapy techniques using adeno-associated viral (AAV) vectors to specifically target microglial cells in the brain. By employing innovative methods such as directed evolution, molecular barcoding, and machine learning, the project aims to enhance the precision of gene delivery to treat various neurological conditions, including Alzheimer's disease and amyotrophic lateral sclerosis. The approach involves using both human and non-human primate models to ensure the effectiveness and safety of the therapies being developed.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for patients suffering from neurodegenerative diseases by specifically targeting harmful inflammation in the brain.
How similar studies have gone: Previous research has shown promise in using AAV vectors for gene therapy, indicating that this approach could lead to significant advancements in treating neurological diseases.
Where this research is happening
-
University of California Berkeley
Berkeley, United States Active
Researchers
- Principal investigator: Schaffer, David V — University of California Berkeley
- Study coordinator: Schaffer, David V
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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.