Cholesterol Pathways Affected by Small Molecules That Decrease Sterol Levels in Niemann-Pick Type C Mutant Cells

Authors: Madalina Rujoi, Nina H. Pipalia, Frederick R. Maxfield

DOI: 10.1371/journal.pone.0012788

Abstract Summary

Researchers identified compounds that reduce cholesterol buildup in Niemann-Pick type C disease, a rare genetic disorder affecting lipid storage. The chemicals work through multiple mechanisms: boosting cholesterol removal from cells, blocking LDL uptake, and increasing cholesteryl ester formation. These findings provide promising leads for developing targeted therapies for this devastating condition.

Why Brain? 🧠

Study identifies compounds that reduce cholesterol buildup in Niemann-Pick type C disease cells by enhancing cholesterol removal, blocking uptake, or altering storage, offering potential therapeutic targets.

The image is AI-generated for illustrative purposes only. Courtesy of Midjourney.