Improving gene delivery to the heart using a new method
Official title: Cardiac AAV gene delivery for clinical translation
Funded at Icahn School of Medicine at Mount Sinai · NIH-11064889 · Data updated 2026-11-12
This study is looking at a new way to improve gene therapy for heart conditions by using a special virus delivered through the heart's blood vessels, which could help the treatment work better and be safer for patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | April 2, 2024 |
| Project end | January 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $736,718 |
| NIH project number | 5R01HL173593-02 |
| Project ID | NIH-11064889 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients with heart failure or other cardiac conditions who may benefit from advanced gene therapy.
✕Less likely to help
Patients with non-cardiac diseases or those who do not have heart-related conditions may not receive any benefit from this research.
What this research studies
This research focuses on enhancing the delivery of gene therapy to the heart using adeno-associated viruses (AAV). The team has developed a novel approach that involves delivering AAV through the coronary arteries while temporarily occluding blood flow, which significantly increases gene expression in heart tissues. By using a catheter-based device to support heart function during the procedure, the method aims to be both safe and effective. The research will explore the factors that contribute to this improved delivery to refine the technique further.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for heart diseases, potentially improving treatment outcomes for patients.
How similar studies have gone: While gene therapy has shown success in non-cardiac applications, this specific approach to cardiac gene delivery is novel and has not been extensively tested in humans.
Where this research is happening
-
Icahn School of Medicine at Mount Sinai
New York, United States Active
Researchers
- Principal investigator: Ishikawa, Kiyotake — Icahn School of Medicine at Mount Sinai
- Study coordinator: Ishikawa, Kiyotake
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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.