Table of Contents

ABSTRACT

Recently, seven gene products have been approved by either the Food and Drug Administration (FDA) or the European Medicines Agency for gene therapy to treat heart failure (HF). Given this rapid pace of progress of gene therapy product development, the FDA has released 6 new guidelines for gene therapy development. Two main approaches for gene therapy treatment are gene therapy gene replacement and gene editing. Replacement of genes involves the provision of a functional copy of a modified gene, while gene editing allows for the correction of existing genetic mutations. There is already an approval for gene therapy for treating genetic disorders like spinal muscular atrophy and Leber’s congenital amaurosis. In cardiovascular diseases (CVDs), research is being conducted to explore the potential role of gene therapy in most developed countries. This communication aims to summarize the mechanisms behind different gene therapies, emphasizing the delivery systems, the associated risk with gene therapy, as well as ongoing clinical trials, and future targets, with a particular emphasis on CVDs.

Keywords: Cardiac damage, cardiac hypertrophy, cardiomyopathy, cardiac dysfunction, gene repair.