Repurposing Dipyridamole in Niemann Pick Type C Disease: A Proof of Concept Study

Niemann Pick type C disease (NPC) is a rare disorder characterized by lysosomal lipid accumulation that damages peripheral organs and the central nervous system. Currently, only miglustat is authorized for NPC treatment in Europe, and thus the identification of new therapies is necessary. The hypoth...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	International journal of molecular sciences 2022-03, Vol.23 (7), p.3456
Hauptverfasser:	Pepponi, Rita, De Simone, Roberta, De Nuccio, Chiara, Visentin, Sergio, Matteucci, Andrea, Bernardo, Antonietta, Popoli, Patrizia, Ferrante, Antonella
Format:	Artikel
Sprache:	eng
Schlagworte:	Adenosine Adenosine - pharmacology Animal models Animals Brain diseases Central nervous system Cholesterol Dipyridamole Dipyridamole - pharmacology Dipyridamole - therapeutic use Disease Drug Repositioning Enzymes Fibroblasts Gene expression Genetic disorders Homeostasis Humans Hypotheses Kinases Lipids Metabolic disorders Metabolism Mice Mitochondria Mutation Niemann-Pick Disease, Type C - pathology Npc1 protein Nucleoside transporter Organs Pathology Phenotypes Proof of Concept Study Proteins
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!