Article
The potential action of galactose as a "chemical chaperone": increase of beta galactosidase activity in fibroblasts from an adult GM1-gangliosidosis patient.
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society - 1 Mar 2009
Caciotti Anna, Donati Maria Alice, d'Azzo Alessandra, Salvioli Rosa, Guerrini Renzo, Zammarchi Enrico, Morrone Amelia
Abstract excerpt
BACKGROUND: The glycosphingolipid storage disorder GM1-gangliosidosis is a severe neurodegenerative condition for which no therapy is currently available. Protein misfolding in lysosomal defects may have the potential to be corrected by chemical chaperones: in vitro and clinical approaches are being investigated. AIMS: We investigated the in vitro effect of galactose on some lysosomal hydrolases, and its in vitro...
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