Ultrasound-activated protein nanostructures for targeted gene delivery
Development of protein-based nanostructures activated by ultrasound
This project develops tiny protein-built bubbles that are opened with ultrasound to deliver gene-editing tools to specific cells, aiming to help people who might need precise, localized gene therapies.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Oklahoma NIH-funded |
| Lab location | 1 site (Norman, United States) |
| Project ID | NIH-11264854 on NIH RePORTER |
What this research studies
The team is making gas vesicle-based protein particles that can carry Cas9 ribonucleoprotein (RNP) complexes and related binding proteins. These particles are designed to form localized “protein clouds” that release their cargo when hit with focused ultrasound. Work includes lab tests in cells and animal models to optimize delivery efficiency, cell viability, and targeting across tissues such as the brain and blood vessels. The goal is a platform that could later be used for safer, more precise gene editing in patients.
Who could benefit from this research
Good fit: People with genetic diseases or conditions that might benefit from localized gene-editing approaches — or patients eligible for early-phase delivery trials of gene therapies — would be the most relevant candidates.
Not a fit: Patients whose conditions require systemic treatments, are not amenable to gene editing, or who are not eligible for early-phase trials are unlikely to benefit directly from this work.
Why it matters
Potential benefit: If successful, this could make gene-editing treatments safer and more precise by delivering therapeutic molecules only where they are needed.
How similar studies have performed: Related approaches using ultrasound or lipid/nanoparticle delivery have shown promise in lab and animal studies, but using gas-vesicle–linked Cas9 for ultrasound-triggered delivery is a novel and early-stage approach.
Where this research is happening
Norman, United States
- University of Oklahoma — Norman, United States (Active)
Researchers
- Principal investigator: Yoon, Sangpil — University of Oklahoma
- Study coordinator: Yoon, Sangpil
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.