732308-64-4Relevant academic research and scientific papers
N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant
Schalli, Michael,Tysoe, Christina,Fischer, Roland,Pabst, Bettina M.,Thonhofer, Martin,Paschke, Eduard,Rappitsch, Tanja,Stütz, Arnold E.,Tschernutter, Marion,Windischhofer, Werner,Withers, Stephen G.
, p. 15 - 22 (2017)
From 1,2;3,4-di-O-isopropylidene-α-D-galactopyranose, a series of highly functionalized (hydroxymethyl)cyclopentanes was easily available. In line with reports by Reymond and J?ger on similar structures, these amine containing basic carbasugars are potent inhibitors of β-D-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase mutant R201C, thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.
