Duke University Researchers Use AAV Gene Therapy to Treat Heart Attack Damage in Primates

DURHAM, N.C., August 28, 2025 – Biomedical engineers at Duke University have conducted a groundbreaking study demonstrating the successful use of gene therapy to treat heart attack damage in non-human primates. The research, published in the journal Circulation Research, is the first of its kind in a non-human primate model of heart disease.

The team, led by Professor Nenad Bursac, used an AAV-based gene therapy that delivers an engineered bacterial sodium channel to the damaged heart. This innovative approach aims to address the permanent damage caused by heart attacks, as heart muscle cells do not naturally regenerate.

Key findings from the study include:

• Improved Function: The gene therapy largely restored the heart’s ability to pump blood and prevented dangerous irregular rhythms (arrhythmia) within weeks of administration.
• Minimal Dosage: The researchers used a dosage 100 times lower than what is clinically approved for similar therapies, and the delivery can be done via a catheter, which avoids the need for open-heart surgery.
• Safety Profile: The genes were not found outside of their intended sites, and no adverse effects were observed during the study period, highlighting a strong safety profile.

This research marks a significant step toward human trials and offers new hope for the hundreds of thousands of Americans who suffer heart attacks each year and are left with a weakened heart.