Synthesis of C-5a-chain extended derivatives of 4-epi-isofagomine: Powerful β-galactosidase inhibitors and low concentration activators of GM1-gangliosidosis-related human lysosomal β-galactosidase.

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

Research output: Contribution to journalArticleResearchpeer-review

Abstract

From an easily available partially protected formal deriv. of 1-deoxymannojirimycin, by hydroxymethyl chain-branching and further elaboration, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have become available. New compds., e.g. I, exhibit improved inhibitory activities comparable to benchmark compd. NOEV (N-octyl-epi-valienamine) and may serve as leads towards improved and more selective pharmacol. chaperones for GM1-gangliosidosis. [on SciFinder(R)]
Original languageEnglish
Pages (from-to)1438-1442
Number of pages5
JournalBioorganic & Medicinal Chemistry Letters
Volume26
Issue number5
DOIs
Publication statusPublished - 2016

Keywords

    Cite this

    Synthesis of C-5a-chain extended derivatives of 4-epi-isofagomine: Powerful β-galactosidase inhibitors and low concentration activators of GM1-gangliosidosis-related human lysosomal β-galactosidase. / Thonhofer, Martin; Weber, Patrick; Santana, Andres Gonzalez; Fischer, Roland; Pabst, Bettina M.; Paschke, Eduard; Schalli, Michael; Stütz, Arnold; Tschernutter, Marion; Windischhofer, Werner; Withers, Stephen G.

    In: Bioorganic & Medicinal Chemistry Letters, Vol. 26, No. 5, 2016, p. 1438-1442.

    Research output: Contribution to journalArticleResearchpeer-review

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    title = "Synthesis of C-5a-chain extended derivatives of 4-epi-isofagomine: Powerful β-galactosidase inhibitors and low concentration activators of GM1-gangliosidosis-related human lysosomal β-galactosidase.",
    abstract = "From an easily available partially protected formal deriv. of 1-deoxymannojirimycin, by hydroxymethyl chain-branching and further elaboration, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have become available. New compds., e.g. I, exhibit improved inhibitory activities comparable to benchmark compd. NOEV (N-octyl-epi-valienamine) and may serve as leads towards improved and more selective pharmacol. chaperones for GM1-gangliosidosis. [on SciFinder(R)]",
    keywords = "imino alditol prepn isofagomine GM1 gangliosidosis galactosidase inhibitor human",
    author = "Martin Thonhofer and Patrick Weber and Santana, {Andres Gonzalez} 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.}",
    note = "M1 - Copyright (C) 2017 American Chemical Society (ACS). All Rights Reserved. CAPLUS AN 2016:171666(Journal; Online Computer File)",
    year = "2016",
    doi = "10.1016/j.bmcl.2016.01.059",
    language = "English",
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    TY - JOUR

    T1 - Synthesis of C-5a-chain extended derivatives of 4-epi-isofagomine: Powerful β-galactosidase inhibitors and low concentration activators of GM1-gangliosidosis-related human lysosomal β-galactosidase.

    AU - Thonhofer, Martin

    AU - Weber, Patrick

    AU - Santana, Andres Gonzalez

    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.

    N1 - M1 - Copyright (C) 2017 American Chemical Society (ACS). All Rights Reserved. CAPLUS AN 2016:171666(Journal; Online Computer File)

    PY - 2016

    Y1 - 2016

    N2 - From an easily available partially protected formal deriv. of 1-deoxymannojirimycin, by hydroxymethyl chain-branching and further elaboration, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have become available. New compds., e.g. I, exhibit improved inhibitory activities comparable to benchmark compd. NOEV (N-octyl-epi-valienamine) and may serve as leads towards improved and more selective pharmacol. chaperones for GM1-gangliosidosis. [on SciFinder(R)]

    AB - From an easily available partially protected formal deriv. of 1-deoxymannojirimycin, by hydroxymethyl chain-branching and further elaboration, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have become available. New compds., e.g. I, exhibit improved inhibitory activities comparable to benchmark compd. NOEV (N-octyl-epi-valienamine) and may serve as leads towards improved and more selective pharmacol. chaperones for GM1-gangliosidosis. [on SciFinder(R)]

    KW - imino alditol prepn isofagomine GM1 gangliosidosis galactosidase inhibitor human

    U2 - 10.1016/j.bmcl.2016.01.059

    DO - 10.1016/j.bmcl.2016.01.059

    M3 - Article

    VL - 26

    SP - 1438

    EP - 1442

    JO - Bioorganic & Medicinal Chemistry Letters

    JF - Bioorganic & Medicinal Chemistry Letters

    SN - 0960-894X

    IS - 5

    ER -