Yonsei Med J.  2024 Oct;65(10):557-571. 10.3349/ymj.2024.0127.

Gene Therapy for Cardiovascular Disease: Clinical Perspectives

• Lee YS
• Choi JR
• Kim JB

• KMID: 2559661
• DOI: http://doi.org/10.3349/ymj.2024.0127

Abstract

Cardiovascular disease (CVD) stands as one of the leading causes of death in the United States, with its prevalence steadily on the rise. Traditional therapeutic approaches, such as pharmacological treatment, cardiovascular intervention, and surgery, have inherent limitations. In response to these challenges, cardiac gene therapy has emerged as a promising alternative for treating CVD patients. However, several obstacles persist, including the low efficiency of gene transduction, immune reactions to vectors or transduced cells, and the occurrence of off-target effects. While preclinical research has demonstrated significant success in various CVD model in both small and large animals, the translation of these findings to clinical applications has, for the most part, yielded disappointing results, except for some early, albeit small, trials. This review aims to provide a comprehensive summary of recent preclinical and clinical studies on gene therapy for various CVDs. Additionally, we discuss the existing limitations and challenges that hinder the widespread clinical application of cardiac gene therapy.

Keyword

Gene therapy; cardiovascular disease; clinical trial