Scientific Breakthrough Aims to Cut LDL Particles by Blocking ApoB Production

Researchers at the Medical University of South Carolina are pursuing a treatment for familial hypercholesterolemia by reducing apoB, the backbone of LDL particles, using human iPSC-derived liver cells to screen about 130,000 compounds. A lead class lowered apoB release and cellular lipid levels in human cells and in humanized mice, bypassing the LDL receptor and potentially helping patients with defective receptors. Further work will clarify the mechanism, safety, and how such drugs could complement existing therapies like statins.