MOLECULAR INTEGRATION AND THERAPEUTIC INNOVATION: THE ROLE OF GENE THERAPY IN THE TREATMENT OF CARDIOVASCULAR DISEASES

Abstract

Gene therapy represents one of the most promising biomedical innovations, allowing the direct modulation of molecular and cellular processes through the introduction of therapeutic genes into somatic cells, with potential for the treatment of cardiovascular diseases refractory to conventional therapies. This study aimed to review and integrate recent scientific evidence on the application of gene therapy in cardiovascular diseases, addressing its mechanisms of action, types of vectors, and clinical perspectives. A narrative and integrative literature review was conducted, searching for articles published between 2019 and 2025 in the PubMed, Scopus, SciELO, and LILACS databases, using descriptors such as "gene therapy," "cardiovascular diseases," and "CRISPR-Cas9," combined with Boolean operators. The analyzed studies demonstrate significant advances in viral vectors and gene editing techniques, such as CRISPR-Cas9 systems, with emphasis on adenoviruses and adeno-associated vectors, which present greater efficiency in gene delivery and improved safety. The main observed applications include the treatment of heart failure, hypertension, and myocardial ischemia, as well as the prevention of restenosis and thrombosis through the modulation of vasodilator and angiogenic genes, such as SERCA2a, VEGF, FGF, and eNOS. Despite encouraging experimental results, clinical trials still face limitations regarding gene expression stability, immunogenicity, and large-scale efficacy. It is concluded that cardiovascular gene therapy constitutes an expanding frontier in precision medicine, with the potential to transform the management of various heart diseases. However, its clinical consolidation depends on the improvement of vectors, the reduction of immunological risks, and the performance of multicenter studies that validate its safety and therapeutic applicability.