Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C.

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

doi:10.1016/j.carres.2016.03.020

Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C.

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

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

Abstract

From an easily available partially protected analog of 1-deoxy-L-gulo-nojirimycin, by chain-branching at C-4 and suitable modification, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have been prepd. New compds. exhibit considerably improved inhibitory activities when compared with the unsubstituted parent compd. and may serve as leads toward new pharmacol. chaperones for GM1-gangliosidosis and Morquio B disease. [on SciFinder(R)]

Original language	English
Pages (from-to)	71-80
Number of pages	10
Journal	Carbohydrate Research
Volume	429
DOIs	https://doi.org/10.1016/j.carres.2016.03.020
Publication status	Published - 2016

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10.1016/j.carres.2016.03.020

Cite this

Thonhofer, M., Weber, P., Gonzalez Santana, A., Tysoe, C., Fischer, R., Pabst, B. M., Paschke, E., Schalli, M., Stütz, A., Tschernutter, M., Windischhofer, W., & Withers, S. G. (2016). Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C. Carbohydrate Research, 429, 71-80. https://doi.org/10.1016/j.carres.2016.03.020

Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C. / Thonhofer, Martin; Weber, Patrick; Gonzalez Santana, Andres et al.
In: Carbohydrate Research, Vol. 429, 2016, p. 71-80.

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

Thonhofer, M, Weber, P, Gonzalez Santana, A, Tysoe, C, Fischer, R, Pabst, BM, Paschke, E, Schalli, M, Stütz, A, Tschernutter, M, Windischhofer, W & Withers, SG 2016, 'Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C.', Carbohydrate Research, vol. 429, pp. 71-80. https://doi.org/10.1016/j.carres.2016.03.020

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title = "Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C.",

abstract = "From an easily available partially protected analog of 1-deoxy-L-gulo-nojirimycin, by chain-branching at C-4 and suitable modification, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have been prepd. New compds. exhibit considerably improved inhibitory activities when compared with the unsubstituted parent compd. and may serve as leads toward new pharmacol. chaperones for GM1-gangliosidosis and Morquio B disease. [on SciFinder(R)]",

keywords = "deoxygulonojirimycin galactosidase inhibitor isofagomine synthesis Morquio imino alditol, human lysosomal galactosidase inhibitor gangliosidosis isofagomine synthesis",

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

year = "2016",

doi = "10.1016/j.carres.2016.03.020",

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journal = "Carbohydrate Research",

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AU - Thonhofer, Martin

AU - Weber, Patrick

AU - Gonzalez Santana, Andres

AU - Tysoe, Christina

AU - Fischer, Roland

AU - Pabst, Bettina M.

AU - Paschke, Eduard

AU - Schalli, Michael

AU - Stütz, Arnold

AU - Tschernutter, Marion

AU - Windischhofer, Werner

AU - Withers, Stephen G.

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AB - From an easily available partially protected analog of 1-deoxy-L-gulo-nojirimycin, by chain-branching at C-4 and suitable modification, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have been prepd. New compds. exhibit considerably improved inhibitory activities when compared with the unsubstituted parent compd. and may serve as leads toward new pharmacol. chaperones for GM1-gangliosidosis and Morquio B disease. [on SciFinder(R)]

KW - deoxygulonojirimycin galactosidase inhibitor isofagomine synthesis Morquio imino alditol

KW - human lysosomal galactosidase inhibitor gangliosidosis isofagomine synthesis

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DO - 10.1016/j.carres.2016.03.020

M3 - Article

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JO - Carbohydrate Research

JF - Carbohydrate Research

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