However, in agreement with the observations from the clinical trials, whether the beneficial effect of lacking ApoC3 relies on the presence of low-density lipoprotein receptor (LDLR) and whether ApoC3 inhibition can be applied for the treatment of severe refractory hypercholesterolemia and atherosclerosis due to LDLR deficiency still needs to be elucidated. The gene discussed is APOC3; the disease is atherosclerosis.