Familial Hypercholesterolemia and Atherosclerosis: Animal Models and Therapeutic Advances

Familial hypercholesterolemia (FH), mainly arising from loss-of-function mutation of the low-density lipoprotein receptor (LDLR), is a life-threatening inherited cardiometabolic disorder with limited therapies. In a recent study, Zhao et al. created a new model of FH and demonstrate that LDLR gene editing protects against both FH and atherosclerosis.