Gene transfer and genome editing for familial hypercholesterolemia
Familial hypercholesterolemia (FH) is an autosomal dominant inherited disease characterized by high circulating low-density lipoprotein (LDL) cholesterol.High circulating LDL cholesterol in FH is due to dysfunctional LDL receptors, and is mainly expressed by hepatocytes.Affected patients rapidly develop atherosclerosis, potentially leading to myoca