Improving heart-targeted gene therapy delivery
Official title: Engineered polymeric adjuvants for heart-redirected targeting and gene therapy
Funded at Case Western Reserve University · NIH-11101109 · Data updated 2026-11-12
This study is looking at new ways to deliver gene therapies directly to the heart to help improve treatments for heart diseases, making them safer and more effective for adults.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Case Western Reserve University NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | June 1, 2024 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $594,531 |
| NIH project number | 5R01HL166241-02 |
| Project ID | NIH-11101109 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults suffering from cardiomyopathies who currently have limited treatment options.
✕Less likely to help
Patients with heart conditions that are not classified as cardiomyopathies may not benefit from this research.
What this research studies
This research focuses on enhancing the delivery of gene therapies specifically to the heart, addressing the significant unmet need for effective treatments for various cardiomyopathies. The approach involves using engineered polymeric adjuvants to improve the targeting and efficacy of therapies like adeno-associated viral (AAV) and nanoparticle (NP) gene therapies. By co-delivering these therapies with synthetic enhancers, the research aims to overcome challenges related to low effectiveness and immune responses in adult patients. The ultimate goal is to increase the safety and effectiveness of these treatments for heart conditions.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients with heart diseases.
How similar studies have gone: Preliminary studies have shown promise in enhancing therapeutic delivery using similar approaches, indicating potential for success.
Where this research is happening
-
Case Western Reserve University
Cleveland, United States Active
Researchers
- Principal investigator: Maiseyeu, Andrei — Case Western Reserve University
- Study coordinator: Maiseyeu, Andrei
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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.