Clinical Efficacy and Safety of Gene Therapy in Cardiovascular Disease: A Systematic Review

Cardiovascular diseases remain the leading cause of mortality worldwide. Most current therapies primarily target managing symptoms and do not address the underlying genetic mechanisms of disease, particularly in inherited and progressive cardiac conditions. In recent years, multiple gene-based therapeutic strategies, including viral and non-viral gene delivery as well as genome editing approaches, have been clinically investigated. The investigations have shown varying degrees of efficacy and safety. However, the available evidence is fragmented across different delivery methods and therapeutic targets, making direct comparison difficult. This systematic review aims to evaluate the clinical efficacy and safety of gene therapy and gene editing approaches in patients with cardiovascular diseases, including heart failure and ischemic heart disease. Furthermore, this review aims to provide a structured overview of the various therapeutic strategies, delivery methods, and study designs employed in different studies. In addition, it aims to compare their outcomes systematically. By integrating and directly comparing the available clinical evidence, this review strives to provide a clear overview of current therapeutic potential, limitations, and differences among gene-based approaches in cardiovascular disease