Gene therapy and genome-editing treatments that can protect patients from coronary artery disease are under investigation
Moataz Dowaidar
Abstract
Globally, the major cause of death is coronary artery disease (CAD). Ethnic groups, nations, or communities are spared. Although millions of people use medication that has been proven to prevent cardiac events, such as statins, ezetimibe and PCSK9 inhibitors, one in two men and one in three women over 40 will have a coronary event throughout their lives. Indeed, after decades of decline, cardiovascular mortality has increased in the US. Even a part of the overlooked events would save billions of dollars in healthcare expenses each year and improve the quality of life for many patients who may otherwise suffer from CAD consequences, including heart failure and arrhythmias. Given the development of CAD as the twenty-first century's most important global health issue, novel preventive agents will be needed.Several innovative drugs that outweigh the traditional pillars of preventive cardiology have either obtained substantial validation in clinical trials, or conducted studies that will be read out in the near future, which is optimistic. Human genetics has predicted the efficiency of many of these medications, with the revelation that naturally existing polymorphisms in critical genes and pathways are linked to CAD protection in individuals fortunate enough to inherit the variants. Additional LDL cholesterol-lowering medications, medications that change other causative lipid characteristics, and anti-inflammatory medications after years of statin, ezetimibe, and PCSK9 inhibitors are the only confirmed effective CAD preventative and therapy regimens. Human genetics supports many of the innovative drugs with genetic studies choosing or validating target genes and pathways. Compared to the prior technique of screening huge libraries of tiny molecules for therapeutic effect, medications may now target specific genes and pathways. Many novel drugs, whether monoclonal antibodies, ASOs, or siRNAs, are biologics. While these medicines provide benefits in terms of efficacy, safety and specificity, they all have short half-lives and are typically administered for the rest of the patient's life. Gene therapies and genome-editing treatments that can protect patients from CAD for the rest of their lives will certainly be produced in the future. Such answers will be vital to the twenty-first century's most critical global health challenge.