The field of gene therapy has experienced significant advancement in the last decade. Originally restricted to experimental biology, gene therapy is now an established clinical modality with demonstrated efficacy in addressing a range of diseases, particularly rare monogenic disorders, hematologic conditions, and oncologic applications. Nevertheless, the cardiovascular system presents both substantial challenges and notable opportunities for innovation in gene therapy. Cardiovascular disease continues to be the leading cause of mortality worldwide. Although advancements have been made in pharmacological treatments, medical devices, and lifestyle modifications, current interventions do not fundamentally address the molecular mechanisms underlying most cardiac diseases. Gene therapy offers distinct potential to modify disease processes at the molecular and cellular level, providing prospects for durable or potentially curative solutions in heart failure, cardiomyopathies, arrhythmias, and vascular pathologies.
RJ Hajjar (Thu,) studied this question.