Breakthrough in Gene Therapy Restores Heart Function in Primates After Heart Attacks
Duke University researchers have successfully used gene therapy to restore heart function in nonhuman primates after heart attacks, paving the way for future human treatments.
Researchers at Duke University have achieved a significant milestone by demonstrating that gene therapy can effectively repair heart damage caused by heart attacks in nonhuman primates. This pioneering study employed an engineered bacterial sodium channel delivered directly to the damaged heart tissue, resulting in notable improvements in heart contraction strength and electrical stability. The therapy successfully restored the heart's ability to pump blood and reduced the occurrence of dangerous arrhythmias within weeks of treatment.
Approximately 800,000 Americans experience a heart attack annually, often leaving the heart with permanent damage. This damage impairs the heart's pumping ability and can lead to heart failure or fatal irregular rhythms. Current treatments primarily slow disease progression but do not restore lost heart function.
In this groundbreaking study, the team first confirmed that the bacterial sodium channels improved contraction in lab-grown human heart tissues. They then successfully applied the gene therapy in macaque monkeys with heart damage replicating human heart attacks. The genes were injected directly into the damaged areas via catheter, with no adverse effects observed. Importantly, the genes remained localized, and the therapy significantly enhanced heart function.
The gene therapy utilizes smaller bacterial genes that can be delivered via viral vectors commonly used in clinical settings, overcoming size limitations of human sodium channel genes. The promising results have generated excitement about potential human trials, with ongoing studies in pigs as the next step towards FDA approval.
This approach offers a dual benefit by improving both electrical and mechanical heart functions and may decrease the risk of arrhythmias—a common complication following heart attacks. Experts believe this therapy could be particularly effective for treating fibrosis and heart failure after myocardial infarction.
Published in Circulation Research, this study marks a pioneering advancement in cardiac gene therapy, paving the way for innovative treatments for heart disease that could eventually transform patient care.
Source: https://medicalxpress.com/news/2025-08-gene-therapy-functionality-nonhuman-primates.html
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