Finding gene mutations that protect against heart attack and developing medicines that mimic them

Sek Kathiresan

Heart attack is now the leading cause of death in the world. However, remarkably few medicines (e.g., aspirin, statins, and antihypertensive agents) are proven to prevent a first heart attack because most medicines fail during the drug development process. I will review an approach researchers are taking to overcome this problem: namely, leveraging the human genome as a tool for prioritizing molecular therapeutic targets for drug development. I will describe an opportunity to leverage the genome’s natural successes by: (1) scouring it for mutations that protect against disease with minimal adverse effects; and (2) developing medicines that mimic them.