Improving how gene therapies reach the brain using focused ultrasound
Improved Delivery of Gene Therapies to the Central Nervous System by Focused Ultrasound-Mediated Disruption of the Blood-Brain Barrier
This study is exploring a new way to help deliver gene therapies to the brain using focused ultrasound, which could make treatments for neurological disorders like Huntington's disease work better and have fewer side effects, so patients might get more effective help in the future.
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-11064795 on NIH RePORTER |
What this research studies
This research focuses on enhancing the delivery of gene therapies to the central nervous system by using focused ultrasound to temporarily disrupt the blood-brain barrier. The goal is to improve the concentration and distribution of adeno-associated viral (AAV) vectors, which are used for treating neurological disorders like Huntington's disease. By combining ultrasound technology with gene therapy, the researchers aim to achieve better therapeutic outcomes while minimizing potential side effects. Patients may benefit from this innovative approach if it proves effective in delivering therapies more efficiently to the brain.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Huntington's disease or other neurological disorders that could benefit from gene therapy.
Not a fit: Patients with conditions unrelated to the central nervous system or those who do not qualify for gene therapy may not receive benefits from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for neurological conditions, improving patient outcomes.
How similar studies have performed: Other research has shown promise in using focused ultrasound for drug delivery across the blood-brain barrier, indicating potential for success in this novel approach.
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.