Cardiovascular disease (CVD), including atherosclerosis and thrombosis, remains a leading cause of death worldwide. The objective of this work is to highlight the potential of RNA-based and gene-editing therapies – specifically small interfering RNAs (siRNAs), antisense oligonucleotides (ASOs), and the CRISPR/Cas9 system – in treating CVD by targeting its genetic causes. These technologies enable precise control of gene expression: SiRNAs and ASOs inhibit the production of harmful proteins at the messenger RNA level, whereas CRISPR/Cas9 allows direct modification of DNA sequences. Such approaches have shown promise in lowering cholesterol, reducing inflammation, and modulating blood clotting factors. Several therapeutics, including inclisiran and pelacarsen, are already in clinical trials. However, safe and efficient delivery remains a key challenge. Advances in delivery platforms – such as lipid nanoparticles, polymers, metallic particles, and plant-derived exosome-like nanoparticles – have enhanced molecular stability and tissue targeting. Notably, GalNAc-conjugates enable precise delivery to the liver. Overall, RNA-based therapies and CRISPR/Cas9 gene editing offer a promising strategy for treating CVD at its genetic root, and ongoing improvements in delivery systems may accelerate their translation into routine clinical care.
Tsouka et al. (Wed,) studied this question.