Crystal structure of human lysosomal acid-alpha-glucosidase, GAA, in complex with iminosugar compound 4i

Crystal structure of human lysosomal acid-alpha-glucosidase, GAA, in complex with iminosugar compound 4i Descriptor: (2~{R},3~{R},4~{R},5~{S})-2-(2-cyclohexylethyl)-2-(hydroxymethyl)piperidine-3,4,5-triol, 1,2-ETHANEDIOL, 2-acetamido-2-deoxy-beta-D-glucopyranose-(1-4)-2-acetamido-2-deoxy-beta-D-glucopyranose, ... Authors: Sulzenbacher, G, Roig-Zamboni, V, Moracci, M, Parenti, G, Py, S. Deposit date: 2024-09-17 Release date: 2025-10-01 Last modified: 2025-10-08 Method: X-RAY DIFFRACTION (2.25 Å) Cite: C -Branched Iminosugars as Selective Pharmacological Chaperones of Lysosomal alpha-Glucosidase for the Treatment of Pompe Disease. J.Med.Chem., 68, 2025

人类溶酶体酸性α-葡萄糖苷酶（GAA）与亚氨基糖（iminosugar）化合物4i结合的晶体结构。描述项：(2R,3R,4R,5S)-2-(2-环己基乙基)-2-(羟甲基)哌啶-3,4,5-三醇、1,2-乙二醇（1,2-ETHANEDIOL）、2-乙酰氨基-2-脱氧-β-D-吡喃葡萄糖（beta-D-glucopyranose）-(1→4)-2-乙酰氨基-2-脱氧-β-D-吡喃葡萄糖（beta-D-glucopyranose）……。作者：Sulzenbacher, G、Roig-Zamboni, V、Moracci, M、Parenti, G、Py, S。沉积日期：2024-09-17；发布日期：2025-10-01；最后修改日期：2025-10-08。研究方法：X射线衍射（X-RAY DIFFRACTION），分辨率2.25埃（Å）。引用文献：《C支链亚氨基糖作为溶酶体α-葡萄糖苷酶的选择性药理分子伴侣用于庞贝病治疗》，《医药化学杂志》（J.Med.Chem.），第68卷，2025年。