LDLR and homozygous familial hypercholesterolemia: Before initiating of phase I/II clinical trial, Greig et al. (2017b) determined the pharmacology and toxicology of clinical candidate vector, AAV8.TBG.hLDLR, as well as those expressed mouse LDLR, AAV8.TBG.mLDLR in a specific mouse model of HoFH.