Targeting MTP for the treatment of homozygous familial hypercholesterolemia

MTP is a protein capable of transferring neutral lipids between membrane vesicles. Its role is essential for the biosynthesis of apolipoprotein B-containing triglyceride-rich lipoproteins. Its function was fully understood after the observation that patients with Abetalipoproteinemia are characterized by mutations in the MTP gene resulting in the loss of its lipid transfer activity. MTP plays also a role in the biosynthesis of CD1, as well as in the regulation of cholesterol ester biosynthesis. In this review, the role of MTP in the assembly of apolipoprotein B is highlighted. Moreover, an overview about the clinical evidence and potentials of MTP inhibition in patients with genetically determined dyslipidemia, namely homozygous familial hypercholesterolemia, will be provided. © 2014 Future Medicine Ltd.