Getting heart gene therapy into the heart using a brief coronary stop‑flow and catheter heart support
Cardiac AAV gene delivery for clinical translation
A new delivery method aims to put gene therapy directly into weakened hearts by briefly directing viral therapy into the coronary vessels while a small catheter pump keeps circulation safe.
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 ID | NIH-11253264 on NIH RePORTER |
What this research studies
If you have heart failure, researchers are developing a way to push gene therapy straight into the heart muscle by briefly stopping flow in the coronary artery and sinus (a "stop‑flow" push). They protect the body’s circulation during that brief period with a catheter‑based heart assist device so the heart does not suffer as the therapy is delivered. In pig heart‑failure models this approach produced much stronger gene expression than standard delivery, even when neutralizing antibodies were present. The team will use large‑animal experiments to pinpoint which mechanical steps boost uptake and to understand how heart cells take up the viral genes so the method can be refined for safe human use.
Who could benefit from this research
Good fit: Ideal candidates for future trials would be people with weakened hearts or heart failure who might benefit from targeted cardiac gene therapy.
Not a fit: People without heart disease, those needing immediate standard treatments, or those who cannot undergo invasive catheter procedures are unlikely to benefit from this preclinical work now.
Why it matters
Potential benefit: If successful, this could enable effective, minimally invasive gene therapy for heart failure by achieving much stronger and safer gene delivery to the heart.
How similar studies have performed: AAV gene therapies have succeeded for non‑heart diseases but have struggled with cardiac uptake; preliminary large‑animal results for this stop‑flow approach show markedly improved delivery, making it promising but not yet tested in humans.
Where this research is happening
New York, United States
- 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
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.