AAV-PCSK9 injection effectively knocked out the LDL receptors (LDLR) in mouse liver as validated by Western blots (Figure S3A–B), and hypercholesterolemia (plasma total cholesterol > 400 mg/dl) was confirmed for hypercholesterolemia alone and d-flow under hypercholesterolemia groups (Figure S3C–G). The gene discussed is LDLR; the disease is familial hypercholesterolemia.