Heart-targeted RNA therapy for weak heart pumping
Cardiac delivery of RNA to treat contractile disorder
This project uses heart-seeking lipid nanoparticles to deliver RNA medicines that aim to correct cellular changes that make the heart pump poorly in people with heart failure.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11321691 on NIH RePORTER |
What this research studies
Researchers are developing special lipid nanoparticles that home to the heart and carry inhibitory RNA medicines (like siRNA and antisense oligonucleotides) to heart muscle cells. They plan to target proteins (VASH1, VASH2, SVBP) that drive harmful changes in the heart's microtubule network and make the heart contract less effectively. Early work will test different RNA cargos and measure how long and how specifically they work in healthy rats before moving to disease models of weak heart function. The goal is to find a safe, lasting RNA approach that can restore better contractile function in hearts affected by these molecular changes.
Who could benefit from this research
Good fit: People with heart failure or recent heart injury who have poor heart pumping or stiffness and who may later qualify for heart-directed RNA therapy trials would be the likely candidates.
Not a fit: Patients whose heart problems are caused by conditions unrelated to the targeted microtubule detyrosination pathway or who cannot receive future delivery methods may not benefit.
Why it matters
Potential benefit: If successful, this approach could improve heart pumping and symptoms in people with heart failure by reversing a specific cellular cause of weakness.
How similar studies have performed: Lipid nanoparticle RNA therapies have proven effective in vaccines and some genetic conditions, but heart-targeted LNPs and blocking VASH/SVBP are largely new and so far shown mainly in animal studies.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Margulies, Kenneth Ber — University of Pennsylvania
- Study coordinator: Margulies, Kenneth Ber
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.