Focused ultrasound to spread gene therapy across brain circuits
Improving Focused Ultrasound Mediated Viral Gene Therapy Delivery
This project aims to use focused ultrasound to carry gene therapy across connected brain regions for conditions like Alzheimer's disease and some movement 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 ID | NIH-11332925 on NIH RePORTER |
What this research studies
Researchers plan to use a method called circuit-focused ultrasound (CIFUS) to follow the brain's natural wiring and deliver viral gene treatments to groups of connected regions in mouse models. They will map white matter connections and apply focused ultrasound to help viral vectors reach those networks. The team will test the approach in mice that model Alzheimer disease with spreading tau pathology and in mouse models of dystonia to see if key disease features improve. Success would help refine the method for possible future human testing.
Who could benefit from this research
Good fit: People with Alzheimer's disease or circuit-based movement disorders (for example certain forms of dystonia) would be the most likely candidates for future clinical trials of this approach.
Not a fit: Patients whose conditions do not involve spreading brain-circuit pathology, those needing immediate treatment now, or those who cannot undergo focused ultrasound procedures may not benefit from this work.
Why it matters
Potential benefit: If successful, this technique could allow targeted gene therapies to reach many affected brain areas and potentially slow or reverse disease-related changes.
How similar studies have performed: Focused ultrasound has been used experimentally to open the blood–brain barrier in humans, but using it to deliver viral gene therapies across connected brain networks is largely novel and has mostly been tested in animals.
Where this research is happening
Dallas, United States
- Ut Southwestern Medical Center — Dallas, United States (Active)
Researchers
- Principal investigator: Shah, Bhavya Ramesh — Ut Southwestern Medical Center
- Study coordinator: Shah, Bhavya Ramesh
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.