When it comes to heart health, lifestyle choices like regular exercise and a balanced diet play a powerful role. Your genes, on the other hand, have been among the variables that have largely been out of our control..
Today, advances in gene therapy are beginning to change that.
As gene therapy advances, inherited genetic risk factors may become treatable through precise, targeted genetic interventions.. Gene therapy works by replacing faulty genes, silencing genes that aren’t functioning properly, or introducing new genes that help restore healthy heart function.
Researchers have long known that genetics plays a significant role in the development of heart disease. As specific genes involved in heart failure and other heart conditions continue to be identified, scientists are uncovering promising targets for gene therapy. It is important to know that many genes are responsible for multiple functions within the body. While progress is moving quickly, researchers are carefully evaluating the safety and long-term effects before these therapies.
Diseases affecting the heart muscles and high cholesterol are among the most enticing targets for future gene therapy. Experts believe gene therapy will offer powerful tools to help better manage, and perhaps prevent, some types of cardiovascular disease.
CVRTI Researchers Advancing Gene Therapy
The Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) researchers are helping lead the way in the gene therapy movement.
In animal models, CVRTIscientists developed an experimental gene therapy aimed at reversing the effects of heart failure and restoring heart function. Heart failure occurs when the heart muscle is unable to pump blood as efficiently as it should. The experimental therapy was able to increase the amount of blood the heart pumps, helping improve survival rates in animal models.
This research focused on a heart protein called cBIN1. Low levels of cBIN1 have been found in heart failure patients, suggesting It plays an important role in maintaining health heart function.The research team aimed to find a way to increase the amount of this protein in animal models.
Researchers inserted an additional copy of the cBIN1 gene into a harmless virus and injected it into the bloodstream.While the findings are promising, further research is needed before human trials can begin.
CAR T Cell Therapy
Another exciting area of research involves CAR T cell therapy for cardiovascular disease. CAR T cell therapy modifies a person’s T cells, which are white blood cells involved in immune response efforts. Researchers are now exploring whether this approach could be adapted to treat cardiovascular conditions, such as acute myocardial infraction (heart attack). When a coronary artery becomes blocked, heart muscle cells can die, increasing the risk of long-term damage.
CAR T, as well as other gene therapies, could be promising approaches for future research to reduce myocardial infarction rates.
Gene Targets for Heart Therapy
In addition to the work on cBIN1, several other genes are being studied as potential targets for heart therapy::
- YAP101: A gene intended to help the heart regenerate its pathways following heart damage. Limited testing in humans has been done,
- S100A1: A protein that has been identified as an enhancer of heart cell function. This could be useful in heart failure with reduced ejection fraction, or when the heart’s left ventricle has trouble pumping. This is considered a potential research target.
- VEGF: Known as vascular endothelial growth factor, this is known to stimulate the growth of new blood vessels to improve blood flow. This could also serve as a marker to observe in relation to risk for heart failure. This line of research has not reached human testing yet.
Searching for Therapy Delivery Methods
One of the biggest challenges in gene therapy is finding the best way to deliver new genes to the correct cells. Researchers commonly use engineered viruses as delivery vehicles. One type that is considered promising is known as adeno-associated virus (AAV) vectors.
AAVs are a popular vector because they are small viruses that are not known to cause disease in humans. Viruses can be engineered to infect a variety of cells, including heart cells, to support future treatment options.
Gene therapy could help reduce the need for surgical treatments. While open-heart surgery carries a level of risk for infections and recovery, gene therapies are designed to be minimally invasive. In some cases, the delivery virus is inserted into the bloodstream. In other cases, a vector is injected directly into a specific tissue.
Future of Gene Therapy
Gene therapy is likely to remain a major focus of cardiovascular research in the coming years.
While the promise is significant, it’s important to remember that medical breakthroughs take time. Years of testing are required to confirm effectiveness and safety of these new therapies in humans. Talk to your healthcare provider if you have questions about a specific gene therapy.
The ultimate goal of gene therapy in heart care is to replace daily medication regimens with one-time, minimally invasive procedures that address disease at its genetic roots. It will take time to fully establish the safety and long-term effectiveness of these treatments, as well as to understand their cost. Current treatments are considered to be expensive and can exceed $1 million per treatment, so future discussions will be essential to determine how cardiovascular gene therapies can be priced and made accessible to patients.
Visit the Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) website to keep up with advances in heart research and gene therapy updates related to cardiovascular health.
