Gene delivery for Alzheimer’s and related brain disorders
Official title: Gene Therapy Delivery for Age-related Neurodegenerative Diseases
Funded at Ut Southwestern Medical Center · NIH-11296841 · Data updated 2026-11-12
This project develops ways to deliver therapeutic genes into the adult brain to help people with Alzheimer’s disease and related tau disorders.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ut Southwestern Medical Center NIH-funded |
| Lab location | 1 site (Dallas, United States) |
| Project start | June 1, 2023 |
| Project end | February 29, 2028 |
| Funding year | 2026 |
| Award (this year) | $712,158 |
| NIH project number | 5R01AG078417-04 |
| Project ID | NIH-11296841 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults aged 21 and older with Alzheimer’s disease or related tauopathies who might be candidates for brain-directed gene therapies would be the intended group for future trials.
✕Less likely to help
People without Alzheimer’s or tau-related neurodegeneration, or those medically ineligible for gene therapy or with pre-existing immunity to AAV9, may not benefit from this work.
What this research studies
This work focuses on getting therapeutic genes into the brain using AAV9 viral vectors and noninvasive focused ultrasound (FUS) to open the blood-brain barrier so a single treatment could reach broad or specific brain regions. The team aims to reduce the total viral dose needed and lower immune risks that have limited adult gene therapy. Research will use laboratory and animal models to test safety, tissue targeting, and durability of gene expression before moving toward human studies. The goal is a delivery approach that is effective across critical brain regions while minimizing invasive surgeries and systemic side effects.
Why it matters
Potential benefit: If successful, this could enable safer, longer-lasting gene treatments that reach large parts of the adult brain and slow or prevent progression of Alzheimer’s and related tauopathies.
How similar studies have gone: AAV9 delivery and focused ultrasound BBB opening have shown promise in animal studies and early human work, but combining these approaches for adult Alzheimer’s remains largely experimental.
Where this research is happening
-
Ut Southwestern Medical Center
Dallas, United States Active
Researchers
- Principal investigator: Bailey, Rachel M — Ut Southwestern Medical Center
- Study coordinator: Bailey, Rachel M
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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.