Gene Editing in Cardiac Disease: A Review of the Literature

Inherited cardiac diseases, including cardiomyopathies and channelopathies, are major contributors to morbidity and sudden cardiac death. Conditions such as long QT syndrome, hypertrophic cardiomyopathy, and dilated cardiomyopathy result from mutations in genes like KCNQ1, KCNH2, and MYBPCF3. X-linked disorders such as danon (lysosomal-associated membrane protein 2) and fabry (generalized lymphatic anomaly) also cause systemic cardiomyopathy. The genetic and phenotypic variability of these disorders highlights the importance of reviewing current diagnostic and treatment strategies. A thorough review of over 40 peer-reviewed articles published between 2010 and 2025 was performed. These included clinical studies, preclinical research, and reviews focusing on genetic mechanisms, disease models, and gene-editing techniques in inherited cardiac conditions. Selected sources emphasized relevance to molecular pathology, therapeutic options, ethical or regulatory issues, minimization, and graft survival. While PSC-CMs and gene editing hold promising therapeutic potential, translating these approaches into human treatments requires improved delivery methods, extensive safety testing, and long-term evaluation. Genome-editing and iPSC technologies are powerful tools for understanding and treating inherited cardiac diseases. Somatic gene editing is generally feasible, whereas germline modifications face ethical and legal obstacles.