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 LDL cholesterol in FH due to dysfunctional receptors, and mainly expressed hepatocytes. Affected patients rapidly develop atherosclerosis, potentially leading myocardial infarction death within the third decade of life if left untreated. Here, we introduce pathogenesis available treatment options. We highlight different possible targets therapeutic intervention. then review gene therapy strategies currently under development, which may become novel options future, discuss their advantages disadvantages. Finally, briefly outline potential applications some these for more common acquired disease.