A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.

Michael Schalli; Patrick Weber; Christina Tysoe; Bettina M. Pabst; Martin Thonhofer; Eduard Paschke; Arnold Stütz; Marion Tschernutter; Werner Windischhofer; Stephen G Withers

doi:10.1016/j.bmcl.2017.05.086

A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.

Michael Schalli, Patrick Weber, Christina Tysoe, Bettina M. Pabst, Martin Thonhofer, Eduard Paschke, Arnold Stütz, Marion Tschernutter, Werner Windischhofer, Stephen G Withers

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

Abstract

N-Functionalized amino(hydroxymethyl)cyclopentanetriols 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 thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.

Original language	English
Pages (from-to)	3431-3435
Journal	Bioorganic & Medicinal Chemistry Letters
Volume	27
Issue number	15
DOIs	https://doi.org/10.1016/j.bmcl.2017.05.086
Publication status	Published - 2017

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10.1016/j.bmcl.2017.05.086

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Schalli, M., Weber, P., Tysoe, C., Pabst, B. M., Thonhofer, M., Paschke, E., Stütz, A., Tschernutter, M., Windischhofer, W., & Withers, S. G. (2017). A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols. Bioorganic & Medicinal Chemistry Letters, 27(15), 3431-3435. https://doi.org/10.1016/j.bmcl.2017.05.086

A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols. / Schalli, Michael; Weber, Patrick; Tysoe, Christina et al.
In: Bioorganic & Medicinal Chemistry Letters, Vol. 27, No. 15, 2017, p. 3431-3435.

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

Schalli, M, Weber, P, Tysoe, C, Pabst, BM, Thonhofer, M, Paschke, E, Stütz, A, Tschernutter, M, Windischhofer, W & Withers, SG 2017, 'A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.', Bioorganic & Medicinal Chemistry Letters, vol. 27, no. 15, pp. 3431-3435. https://doi.org/10.1016/j.bmcl.2017.05.086

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abstract = "N-Functionalized amino(hydroxymethyl)cyclopentanetriols 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 thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.",

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

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AU - Weber, Patrick

AU - Tysoe, Christina

AU - Pabst, Bettina M.

AU - Thonhofer, Martin

AU - Paschke, Eduard

AU - Stütz, Arnold

AU - Tschernutter, Marion

AU - Windischhofer, Werner

AU - Withers, Stephen G

PY - 2017

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AB - N-Functionalized amino(hydroxymethyl)cyclopentanetriols 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 thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.

U2 - 10.1016/j.bmcl.2017.05.086

DO - 10.1016/j.bmcl.2017.05.086

M3 - Article

SN - 0960-894X

VL - 27

SP - 3431

EP - 3435

JO - Bioorganic & Medicinal Chemistry Letters

JF - Bioorganic & Medicinal Chemistry Letters

IS - 15

ER -

A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.

Abstract

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