The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidaseResearch in context

Background: Gaucher Disease is caused by mutations of the GBA gene which encodes the lysosomal enzyme acid beta-glucosidase (GCase). GBA mutations commonly affect GCase function by perturbing its protein homeostasis rather than its catalytic activity. Heat shock proteins are well known cytoprotectiv...

Full description

Bibliographic Details
Main Authors: Cathrine K. Fog, Paola Zago, Erika Malini, Lukasz M. Solanko, Paolo Peruzzo, Claus Bornaes, Raffaella Magnoni, Arnela Mehmedbasic, Nikolaj H.T. Petersen, Bruno Bembi, Johannes F.M.G. Aerts, Andrea Dardis, Thomas Kirkegaard
Format: Article
Language:English
Published: Elsevier 2018-12-01
Series:EBioMedicine
Online Access:http://www.sciencedirect.com/science/article/pii/S2352396418305401