TY - JOUR
T1 - N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant.
AU - Schalli, Michael
AU - Tysoe, Christina
AU - Fischer, Roland
AU - Pabst, Bettina M.
AU - Thonhofer, Martin
AU - Paschke, Eduard
AU - Rappitsch, Tanja
AU - Stütz, Arnold
AU - Tschernutter, Marion
AU - Windischhofer, Werner
AU - Withers, Stephen G
N1 - M1 - Copyright (C) 2017 American Chemical Society (ACS). All Rights Reserved.
CAPLUS AN 2017:454307(Journal; Online Computer File)
PY - 2017
Y1 - 2017
N2 - 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 Jager on similar structures, these amine contg. basic carbasugars are potent inhibitors of β-D-galactosidases and, for the first time, could be shown to act as pharmacol. chaperones for GM1-gangliosidosis-assocd. lysosomal acid β-galactosidase mutant R201C, thus representing a new structural type of pharmacol. chaperones for this lysosomal storage disease. [on SciFinder(R)]
AB - 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 Jager on similar structures, these amine contg. basic carbasugars are potent inhibitors of β-D-galactosidases and, for the first time, could be shown to act as pharmacol. chaperones for GM1-gangliosidosis-assocd. lysosomal acid β-galactosidase mutant R201C, thus representing a new structural type of pharmacol. chaperones for this lysosomal storage disease. [on SciFinder(R)]
KW - crystal mol structure dioxolo cyclopenta isoxazolol
KW - isopropylidenedioxy cyclopenta isoxazole crystal mol structure
KW - amino hydroxymethyl cyclopentanetriol prepn pharmacol chaperone gangliosidosis
KW - human lysosomal galactosidase amino hydroxymethyl cyclopentanetriol pharmacol chaperone
U2 - 10.1016/j.carres.2017.03.009
DO - 10.1016/j.carres.2017.03.009
M3 - Artikel
SN - 0008-6215
VL - 443-444
SP - 15
EP - 22
JO - Carbohydrate Research
JF - Carbohydrate Research
ER -