Gene therapy for treating age-related brain diseases
Official title: Gene Therapy Delivery for Age-related Neurodegenerative Diseases
Funded at Ut Southwestern Medical Center · NIH-11039966 · Data updated 2026-11-12
This study is looking at a new way to help people with Alzheimer's by using a special kind of gene therapy that delivers helpful genes directly to brain cells, aiming to provide lasting benefits with just one treatment and without the need for invasive procedures.
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 | 2025 |
| Award (this year) | $700,777 |
| NIH project number | 5R01AG078417-03 |
| Project ID | NIH-11039966 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals aged 21 and older who are experiencing symptoms of age-related neurodegenerative diseases, particularly those related to abnormal tau protein.
✕Less likely to help
Patients with non-neurodegenerative conditions or those under 21 years old may not receive any benefit from this research.
What this research studies
This research focuses on using gene therapy to address age-related neurodegenerative diseases like Alzheimer's. It employs adeno-associated virus (AAV) vectors to deliver therapeutic genes directly to brain cells, aiming for both targeted and widespread treatment. The approach seeks to overcome challenges associated with traditional delivery methods, such as invasive procedures and immune responses, by exploring non-invasive techniques to enhance gene delivery to the brain. Patients may benefit from a long-lasting intervention from a single treatment.
Why it matters
Potential benefit: If successful, this research could provide a novel treatment option that significantly slows or halts the progression of neurodegenerative diseases.
How similar studies have gone: Previous research using AAV vectors for neurological disorders has shown promising results, indicating potential for success in this novel approach.
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.