Using focused ultrasound to help gene therapies reach more of the brain
Improved Delivery of Gene Therapies to the Central Nervous System by Focused Ultrasound-Mediated Disruption of the Blood-Brain Barrier
This project uses focused ultrasound to help gene therapies get across the blood-brain barrier and reach more brain areas for people with Huntington's disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11305282 on NIH RePORTER |
What this research studies
From a patient's point of view, the team is trying to temporarily open the blood-brain barrier with MRI-guided focused ultrasound so intravenous gene vectors can enter the brain at higher concentrations and over larger areas. Right now, IV delivery spreads widely but at low doses, and direct brain injections give high doses only near the injection site; this approach aims to combine the best of both. The researchers will develop and test delivery methods using AAV-packaged micro-RNA designed to lower the mutant Huntingtin gene that causes Huntington's disease, building on early lab results that show better delivery after ultrasound opening. The work is mainly preclinical but is meant to lead toward safer, more effective brain-directed gene therapies.
Who could benefit from this research
Good fit: The primary candidates for future trials would be people with genetically confirmed Huntington's disease who meet safety criteria for MRI-guided focused ultrasound and gene therapy.
Not a fit: People without Huntington's disease or those who cannot undergo MRI or ultrasound-guided procedures are unlikely to benefit from this specific line of work.
Why it matters
Potential benefit: If successful, this could allow gene therapies to reach affected brain regions more reliably and at effective doses, potentially improving outcomes and reducing side effects for people with Huntington's disease.
How similar studies have performed: Focused ultrasound has opened the blood-brain barrier safely in early human trials and improved AAV delivery in animal studies, but combining FUS with AAV gene therapy for Huntington's remains largely at the preclinical stage.
Where this research is happening
Boston, United States
- Brigham and Women's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Todd, Nicholas E. — Brigham and Women's Hospital
- Study coordinator: Todd, Nicholas E.
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.