Improving gene delivery to the heart using a new method

Cardiac AAV gene delivery for clinical translation

NIH-funded research Icahn School of Medicine at Mount Sinai · NIH-11064889

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 typeR01 grant
Study typeNIH-funded research
Funding institutionIcahn School of Medicine at Mount Sinai NIH-funded
Lab location1 site (New York, United States)
Project IDNIH-11064889 on NIH RePORTER

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.

Who could benefit from this research

Good fit: Ideal candidates for this research are patients with heart failure or other cardiac conditions who may benefit from advanced gene therapy.

Not a fit: Patients with non-cardiac diseases or those who do not have heart-related conditions may not receive any benefit from this research.

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 performed: 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

New York, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.