Apolipoprotein B-100 (ApoB-100)

Apolipoprotein B-100 is a large, non-exchangeable structural protein essential for the assembly, secretion, and metabolism of triglyceride-rich lipoproteins such as VLDL and LDL. It is encoded by the APOB gene, which produces a single large mRNA transcript in the liver; this mRNA is translated into ApoB-100. ApoB-100 serves as the critical ligand for the LDL receptor, mediating cellular uptake of LDL particles, and is thus a central player in cholesterol homeostasis and atherogenesis[2][5][7]. Antisense drugs targeting ApoB-100 mRNA (such as mipomersen) reduce ApoB-100 synthesis, leading to lower LDL-C levels in the blood, but may present safety concerns such as hepatic steatosis[2]. Note: The target name "Apolipoprotein B100 mRNA" refers to the mRNA transcript coding for apolipoprotein B-100, not the protein itself. While the mRNA is a valid therapeutic target for antisense oligonucleotides, "Apolipoprotein B-100" is the canonical name for the actual protein. Thus, using "Apolipoprotein B100 mRNA" as a general biological target is not entirely correct; rather, it is more accurate to list the target as "Apolipoprotein B-100" (ApoB-100), with a focus on mRNA inhibition as a mechanism of action. Therefore, is_incorrect: true because the submitted target name does not match canonical naming conventions and does not refer to a conventional protein, receptor, or enzyme but rather its mRNA.

Other names

Apolipoprotein BApoBApolipoprotein B100ApoB100

Inhibition of ApoB-100 mRNA translation (e.g., by antisense oligonucleotides like mipomersen[2]); Inhibition of ApoB lipidation and VLDL assembly (e.g., by MTP inhibitors like lomitapide[6]); Increased clearance of ApoB-containing lipoproteins (indirect mechanism of statins/PCSK9 inhibitors)