The phase I and II trials of AAV‐based liver‐directed AAV8.TBG.hLDLR aiming to assess the safety and efficacy of the treatment for HoFH are currently underway (NCT02651675; AAV8‐mediated LDLR Gene Replacement in Subjects With HoFH). Here, LDLR is linked to homozygous familial hypercholesterolemia.