INVESTIGATION OF DIPIRYDAMOLE-ELICITED SIGNALING IN THE BRAIN OF NIEMANN PICK TYPE C MICE: A MULTI-OMIC STUDY.

Niemann Pick type C1 (NPC1) is a rare, fatal disorder characterized by endosomal lipid accumulation that leads to damage of both peripheral organs and central nervous system (cerebellum and hippocampus are especially affected). Currently, miglustat is the only approved drug for NPC1, thus the identification of new treatments is mandatory. We have previously demonstrated that the drug dipyridamole (DIP), an approved medicine that is clinically employed as an antiplatelet agent, rescued recognition memory and increased hippocampal expression of calbindin. On the contrary, the drug was unable to improve cerebellar-dependent motor function. In order to understand the mechanism of action engaged by DIP to exert its protective action in the hippocampus, we performed a multi-omic analysis of gene and proteins modulated by the treatment in the hippocampus and cerebellum of a mouse model of NPC1 disease.