Ultrasound-assisted gene delivery to the heart
Ultrasound Cavitation for Facilitated Cardiac Transduction of AAV
This project uses ultrasound plus tiny gas-filled bubbles to help AAV gene therapy reach heart muscle cells so people with heart disease could get lower doses and fewer side effects.
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-11378485 on NIH RePORTER |
What this research studies
Researchers are using diagnostic ultrasound together with microbubbles to create brief cavitation that helps adeno-associated virus (AAV) gene vectors enter cardiomyocytes. Early lab and animal work shows this method can markedly increase AAV9 uptake by heart muscle cells even when the virus is not attached to the bubbles. The aim is to raise delivery efficiency so effective therapy can be given at much lower viral doses, reducing the risk of dose-related immune reactions. The work is being developed at the University of Virginia as a preclinical pathway toward future human testing.
Who could benefit from this research
Good fit: People with inherited or acquired heart muscle diseases who might be candidates for AAV-based heart gene therapy would be the intended candidates for future trials.
Not a fit: People without heart disease, those not eligible for gene therapy, or individuals with pre-existing strong immunity to AAV are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this could make heart-directed AAV gene therapies safer and effective at much lower viral doses.
How similar studies have performed: AAV9 has shown strong heart-targeting in preclinical studies but required high doses that caused immune problems in early human trials, while ultrasound plus microbubble delivery has increased DNA delivery in prior work and shows promising pilot results for improving AAV cardiac transduction.
Where this research is happening
Charlottesville, United States
- University of Virginia — Charlottesville, United States (Active)
Researchers
- Principal investigator: Lindner, Jonathan R — University of Virginia
- Study coordinator: Lindner, Jonathan R
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.