Apolipoprotein B (APOB) (APOB)

Apolipoprotein B (ApoB) is a critical structural protein found in all atherogenic lipoproteins, including very-low-density lipoprotein (VLDL) and low-density lipoprotein (LDL) [1, 2]. The human APOB gene, located on chromosome 2, encodes two primary isoforms: ApoB-100, synthesized in the liver, and ApoB-48, synthesized in the intestine [1, 2]. ApoB-100 is essential for the assembly and secretion of VLDL and serves as the primary ligand for the LDL receptor, facilitating the clearance of cholesterol from the blood [2, 3]. Mutations in the APOB genomic DNA are a major cause of familial hypercholesterolemia and hypobetalipoproteinemia [1]. Pharmacological targeting of ApoB includes the antisense oligonucleotide mipomersen, which binds to APOB mRNA to inhibit protein translation, thereby reducing circulating levels of atherogenic particles [4]. While effective in lowering LDL-C, direct inhibition of ApoB synthesis is associated with safety concerns such as hepatic steatosis due to the accumulation of lipids in the liver [3, 5].

Other names

Apo BApoB-100ApoB-48Apolipoprotein B-100APOB gene

Antisense oligonucleotide-mediated degradation of mRNA to inhibit protein synthesis; potential target for gene editing.