N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant.

Michael Schalli; Christina Tysoe; Roland Fischer; Bettina M. Pabst; Martin Thonhofer; Eduard Paschke; Tanja Rappitsch; Arnold Stütz; Marion Tschernutter; Werner Windischhofer; Stephen G Withers

doi:10.1016/j.carres.2017.03.009

N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant.

Michael Schalli, Christina Tysoe, Roland Fischer, Bettina M. Pabst, Martin Thonhofer, Eduard Paschke, Tanja Rappitsch, Arnold Stütz^*, Marion Tschernutter, Werner Windischhofer, Stephen G Withers

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

Abstract

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)]

Original language	German
Pages (from-to)	15-22
Number of pages	8
Journal	Carbohydrate Research
Volume	443-444
DOIs	https://doi.org/10.1016/j.carres.2017.03.009
Publication status	Published - 2017

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10.1016/j.carres.2017.03.009

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Schalli, M., Tysoe, C., Fischer, R., Pabst, B. M., Thonhofer, M., Paschke, E., Rappitsch, T., Stütz, A., Tschernutter, M., Windischhofer, W., & Withers, S. G. (2017). N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant. Carbohydrate Research, 443-444, 15-22. https://doi.org/10.1016/j.carres.2017.03.009

Schalli, M, Tysoe, C, Fischer, R, Pabst, BM, Thonhofer, M, Paschke, E, Rappitsch, T, Stütz, A, Tschernutter, M, Windischhofer, W & Withers, SG 2017, 'N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant.', Carbohydrate Research, vol. 443-444, pp. 15-22. https://doi.org/10.1016/j.carres.2017.03.009

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title = "N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant.",

abstract = "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)]",

keywords = "crystal mol structure dioxolo cyclopenta isoxazolol, isopropylidenedioxy cyclopenta isoxazole crystal mol structure, amino hydroxymethyl cyclopentanetriol prepn pharmacol chaperone gangliosidosis, human lysosomal galactosidase amino hydroxymethyl cyclopentanetriol pharmacol chaperone",

author = "Michael Schalli and Christina Tysoe and Roland Fischer and Pabst, {Bettina M.} and Martin Thonhofer and Eduard Paschke and Tanja Rappitsch and Arnold St{\"u}tz and Marion Tschernutter and Werner Windischhofer and Withers, {Stephen G}",

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doi = "10.1016/j.carres.2017.03.009",

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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

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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

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DO - 10.1016/j.carres.2017.03.009

M3 - Artikel

SN - 0008-6215

VL - 443-444

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JO - Carbohydrate Research

JF - Carbohydrate Research

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