Discovery and validation of new molecular targets in treating dyslipidemia: the role of human genetics

AV Khera, DJ Rader - Trends in Cardiovascular Medicine, 2009 - Elsevier
Several high-profile failures of lipid-related therapeutics in clinical trials have led to intense
interest in improved discovery and preclinical prioritization of potential targets. The careful
study of patients with rare monogenic disorders has played a key role in establishing the
causal role of cholesterol in atherosclerosis and highlighting viable drug targets. Systematic
efforts to extend the association of common variants linked with lipid levels to coronary
disease enable assessment of the vascular consequences of lifelong differences in lipids …