We previously reported the development and evaluation of an AAV8 vector expressing a gain-of-function, human LDLR triple-mutant variant (hLDLR-L318D/K809R/C818A) in which this LDLR variant exhibited partial resistance to degradation via PCSK9 and IDOL pathways in mouse models of LDLR deficiency.24 This evidence concerns the gene LDLR and hyperinsulinemic hypoglycemia, familial, 4.