Enhancing gene therapy delivery for brain diseases using focused ultrasound
Official title: Improving Focused Ultrasound Mediated Viral Gene Therapy Delivery
Funded at Ut Southwestern Medical Center · NIH-11094841 · Data updated 2026-11-12
This study is exploring a new way to deliver gene therapy to the brain using a special ultrasound technique, which could help improve treatments for conditions like Alzheimer's disease by better targeting the areas of the brain that need it most.
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, 2024 |
| Project end | April 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $811,897 |
| NIH project number | 5R01NS127900-02 |
| Project ID | NIH-11094841 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would be individuals with neurodegenerative diseases, particularly those with Alzheimer's disease or related conditions.
✕Less likely to help
Patients with non-neurological conditions or those who do not have access to advanced gene therapy options may not benefit from this research.
What this research studies
This research aims to improve the delivery of gene therapy to the brain by using a technique called circuit focused ultrasound (CIFUS). By mapping brain connectivity, the researchers plan to target interconnected brain regions more effectively, which could help overcome current limitations in gene therapy for conditions like Alzheimer's disease. The study will utilize mouse models to test this innovative approach, focusing on diseases that affect brain circuits and function. If successful, this method could significantly enhance the distribution of therapeutic gene products throughout the central nervous system.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for brain diseases by improving the delivery of gene therapies.
How similar studies have gone: Previous research has shown promise in using focused ultrasound for drug delivery, but this specific approach is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Ut Southwestern Medical Center
Dallas, United States Active
Researchers
- Principal investigator: Shah, Bhavya Ramesh — Ut Southwestern Medical Center
- Study coordinator: Shah, Bhavya Ramesh
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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.