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

Research output: Contribution to journalArticleResearchpeer-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 languageGerman
Pages (from-to)15-22
Number of pages8
JournalCarbohydrate Research
Volume443-444
DOIs
Publication statusPublished - 2017

Keywords

    Cite this

    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; Tschernutter, Marion; Windischhofer, Werner; Withers, Stephen G.

    In: Carbohydrate Research, Vol. 443-444, 2017, p. 15-22.

    Research output: Contribution to journalArticleResearchpeer-review

    Schalli, Michael ; Tysoe, Christina ; Fischer, Roland ; Pabst, Bettina M. ; Thonhofer, Martin ; Paschke, Eduard ; Rappitsch, Tanja ; Stütz, Arnold ; Tschernutter, Marion ; Windischhofer, Werner ; Withers, Stephen G. / N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant. In: Carbohydrate Research. 2017 ; Vol. 443-444. pp. 15-22.
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    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)]",
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    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}",
    note = "M1 - Copyright (C) 2017 American Chemical Society (ACS). All Rights Reserved. CAPLUS AN 2017:454307(Journal; Online Computer File)",
    year = "2017",
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    language = "deutsch",
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    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

    VL - 443-444

    SP - 15

    EP - 22

    JO - Carbohydrate Research

    JF - Carbohydrate Research

    SN - 0008-6215

    ER -