Using ultrasound to edit genes in the blood-brain barrier
Genome Editing the Blood-Brain Barrier with Sonoselective Focused Ultrasound
This study is exploring a new way to help medicines reach the brain better by using ultrasound to change certain cells in the blood-brain barrier, which could make treatments for brain-related conditions more effective and safer.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Virginia NIH-funded |
| Lab location | 1 site (Charlottesville, United States) |
| Project ID | NIH-10796779 on NIH RePORTER |
What this research studies
This research investigates a novel method to enhance drug delivery to the brain by using ultrasound to edit genes in the blood-brain barrier (BBB). The approach focuses on safely targeting endothelial cells, which form the barrier, to improve the effectiveness of treatments for central nervous system disorders. By employing a technique called sonoselective gene delivery, researchers aim to overcome challenges posed by efflux transporters that limit drug exposure in the brain. This method is guided by MRI imaging to ensure precise targeting and minimize invasiveness.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals suffering from central nervous system disorders that require improved drug delivery methods.
Not a fit: Patients with conditions unrelated to central nervous system disorders may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve the delivery of therapeutic agents to the brain, enhancing treatment outcomes for patients with neurological disorders.
How similar studies have performed: While the concept of using ultrasound for gene delivery is emerging, this specific approach targeting the blood-brain barrier is relatively novel and has not been extensively tested in prior studies.
Where this research is happening
Charlottesville, United States
- University of Virginia — Charlottesville, United States (Active)
Researchers
- Principal investigator: Price, Richard J. — University of Virginia
- Study coordinator: Price, Richard J.
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.