Advances in targeted nucleic acid therapies for the prevention of cardiovascular diseases

Nucleic acid-based therapies represent a major breakthrough in modern medicine, especially in preventing and treating cardiovascular diseases. These innovative approaches, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and advanced gene-editing technologies such as CRISPR/Cas9, offer new ways to overcome the limitations of conventional drug therapies.

Unlike traditional drugs, nucleic acid-based therapies enable precise molecular-level interventions, targeting specific genes responsible for disease development. These technologies can modulate key molecular targets, including proprotein convertase subtilisin/kexin type 9 (PCSK9), apolipoprotein(a), angiopoietin-like 3 (ANGPTL3), apolipoprotein C-III (APOC3), and angiotensinogen, all of which are critical in lipid metabolism and blood pressure regulation.

Clinical trials are already showing encouraging results. These RNA- and DNA-based approaches have the potential to improve therapy efficacy significantly while enhancing patient adherence. For patients unresponsive to traditional treatments, these options could be a major step forward in care. A key advantage is their infrequent administration, with some therapies requiring only biannual doses. Particularly noteworthy is their ability to effectively lower low-density lipoprotein (LDL) cholesterol, crucial for patients with genetic lipid metabolism disorders like familial hypercholesterolemia. They also reduce the production of hard-to-treat proteins like lipoprotein(a), which plays a major role in atherosclerosis development.

Our study provides a comprehensive overview of these advances, offering insights into the mechanisms driving these therapies. It highlights their potential to transform cardiovascular medicine by augmenting existing therapies and improving prevention strategies for serious cardiovascular diseases.