Advances in research on potential therapeutic approaches for Niemann-Pick C1 disease

C Zhang, K Su, X Jiang, Y Tian, K Li - Frontiers in Pharmacology, 2024 - frontiersin.org
Niemann-Pick disease type C1 (NP-C1) is a rare and devastating recessive inherited
lysosomal lipid and cholesterol storage disorder caused by mutations in the NPC1 or NPC2 …

Structural and biochemical analysis of ligand binding in yeast Niemann–Pick type C1–related protein

L Nel, K Thaysen, D Jamecna, E Olesen… - Life Science …, 2025 - life-science-alliance.org
In eukaryotes, integration of sterols into the vacuolar/lysosomal membrane is critically
dependent on the Niemann–Pick type C (NPC) system. The system consists of an integral …