Gene Therapy: A Paradigm Shift In The Treatment Of Heart Rhythm Disorders
Cardiovascular disease or heart disease is the leading cause of death worldwide, accounting for over 30% of all global deaths. Despite advances in modern medicine, there are still unmet needs in cardiovascular disease care. Gene therapy has emerged as the most promising option in recent years and could play an important role in the treatment of heart conditions such as severe cardiac and peripheral ischemia, heart arrhythmias, heart failure, and so on. The vital role of genetic factors in human health is now well-established as a result of accelerated research and the rapid development of effective gene-editing technology.
While cardiovascular gene therapy is a game changer, there are two developments of particular interest as they have the potential to transform cardiac care.
Gene Therapy For Heart Rhythm Disorders
Heart rhythm disorders or arrhythmias are extremely common and may not always be threatening. However, some abnormal heart rhythms can be life-threatening and are linked to sudden cardiac arrest, stroke, and heart failure. Heart arrhythmias may also develop as a result of some types of heart disease. One of the most troubling forms of arrhythmia remains arrhythmogenic cardiomyopathy, which is a genetic heart disease. This is regarded as the main cause of sudden cardiac arrest in younger adults, including athletes aged 35 years and under.
Patients with arrhythmogenic cardiomyopathy are not born with any heart defects or abnormalities, but they develop irregularities in their heartbeat during their 20s or 30s. This can lead to a dangerous rise in heart rate, which is why it causes sudden cardiac arrest in some individuals who appear to be otherwise healthy. For this reason, patients with the condition are advised against intensive or prolonged exercise. However, current treatments have been limited to using an implantable defibrillator, but the disease progresses until a heart transplant becomes necessary.
With new gene therapy, researchers will finally be able to address the underlying genetic cause of arrhythmogenic cardiomyopathy. Genetic studies of the disease’s pathology have revealed that a protein known as Connexin 43 facilitates communication between cells in healthy hearts. Although Connexin 43 levels are normal in diseased hearts, the protein was not present in the required locations. This is because of inadequate production of a protein called GJA1-20k, which is necessary for the trafficking of Connexin 43. With low doses of gene therapy, researchers have been able to restore GJA1-20k protein levels, which in turn allowed cells of the heart to transport Connexin 43 to the appropriate locations, restoring a more normal heartbeat.
These gene therapy developments have broader implications as it means that researchers have now found a way to stabilize cardiac electrical activity effectively and safely. It also means that there may be a potential gene therapy to treat dangerous arrhythmias that are linked to common conditions, including rhythm disorders that develop after a heart attack.
Although ion channel-blocking drugs that are widely used to treat arrhythmias are life-saving for patients, they can give rise to new rhythm disorders and slow the heart in some patients. Gene therapy would offer an effective and safe alternative that addresses the underlying problem.
Recommended Read: Pan-Aortic Replacement Surgery: Advancements, Challenges, and Outcomes in Treating Extensive Aortic Disease
References:
