The effects of Cypor deficiency on lipid metabolism and cholesterol clearance could not be compensated by unedited hepatocytes in the livers of Ldlr−/− mice, indicating that while this approach was effective in the PKU mouse model, transplantation of Cypor-deficient hepatocytes is not suitable for treating HoFH. Here, LDLR is linked to homozygous familial hypercholesterolemia.