Using RNA delivery to improve heart function in patients with contractile disorders
Cardiac delivery of RNA to treat contractile disorder
['FUNDING_R01'] · UNIVERSITY OF PENNSYLVANIA · NIH-11117128
This study is testing a new way to deliver helpful RNA directly to the heart using tiny particles, aiming to improve heart function for people with conditions that make it hard for their heart to pump effectively.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PENNSYLVANIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11117128 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a new method to deliver therapeutic RNA directly to the heart to treat contractile disorders, which affect the heart's ability to pump effectively. The approach utilizes specially designed lipid nanoparticles that can efficiently transport RNA molecules to cardiac cells. By targeting specific proteins involved in heart function, the goal is to improve heart contractility and overall cardiac health. Patients may be involved in studies that assess the effectiveness of these treatments in improving heart function.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with heart failure or other conditions that impair heart contractility.
Not a fit: Patients with stable heart function or those not experiencing contractile dysfunction may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve heart function in patients with contractile disorders.
How similar studies have performed: Other research has shown promise in using lipid nanoparticles for RNA delivery, particularly in the context of mRNA vaccines, suggesting a potential for success in this novel application.
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.