Heart disease remains a global epidemic, accounting for approximately 32% of all deaths each year. The most common type of heart disease is coronary artery disease, a condition in which blood flow to the heart is impeded, potentially leading to a heart attack if left untreated. In an effort to tackle this pervasive issue, researchers from the University of Texas Southwestern Medical Center have made a groundbreaking discovery with their new CRISPR-Cas9 gene editing therapy.
According to Eric N. Olson, Ph.D., Professor and Chairman of the Department of Molecular Biology at the University of Texas Southwestern Medical Center, the limitations of current treatment options for heart disease make it imperative to explore new solutions. In a recent study published in the journal Science, Dr. Olson and his team found that the CRISPR-Cas9 gene editing system, packaged into a viral delivery system that targets the heart in mice and large mammals, holds the potential to both treat heart disease and repair damaged tissue following a heart attack.
The gene editing system, composed of a guide RNA and a base editor, enables researchers to target disease mediators with precision, potentially leading to a higher therapeutic benefit with fewer adverse side effects. When asked about the potential impact of this research, Dr. Olson stated that although a lot of work remains to be done, the approach could eventually be used to improve cardiac function after a heart attack. The next steps in this research include improving the efficiency and specificity of the CRISPR-Cas9 gene editing construct, as well as finding alternative, non-viral-based delivery methods.
The potential of this gene therapy has not gone unnoticed, as it has been dubbed a “game changer” by Dr. Richard Wright, a cardiologist at Providence Saint John’s Health Center in Santa Monica, CA. Dr. Wright acknowledged that while the study was conducted in mice, and not all therapies that work for mice will work for humans, the ability to manipulate the body’s response to injury holds enormous potential. However, Dr. Wright also warned that there are still safety studies to be performed and the technology still needs to be translated into larger animals, including pigs and humans, to determine its safety and efficacy.