Synthesis of a New β-Galactosidase Inhibitor Displaying Pharmacological Chaperone Properties for GM1 Gangliosidosis

GM1 gangliosidosis is a rare lysosomal disease caused by the deficiency of the enzyme β-galactosidase (β-Gal; <i>GLB1</i>; E.C. 3.2.1.23), responsible for the hydrolysis of terminal β-galactosyl residues from GM1 ganglioside, glycoproteins, and glycosaminoglycans, such as keratan-sulfate...

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Bibliographic Details

Published in:	Molecules
Main Authors:	Francesca Clemente, Macarena Martínez-Bailén, Camilla Matassini, Amelia Morrone, Silvia Falliano, Anna Caciotti, Paolo Paoli, Andrea Goti, Francesca Cardona
Format:	Article
Language:	English
Published:	MDPI AG 2022-06-01
Subjects:	iminosugars β-galactosidase inhibitors GM1 gangliosidosis <i>GLB1</i> pharmacological chaperones nitrones
Online Access:	https://www.mdpi.com/1420-3049/27/13/4008