Division of Biological Chemistry and Drug Discovery, College of Life Sciences, University of Dundee, Dow Street, Dundee, DD1 5EH, UK.
Org Biomol Chem. 2014 Mar 28;12(12):1919-34. doi: 10.1039/c3ob42164c. Epub 2014 Feb 12.
A series of synthetic analogues of 1-D-(2-amino-2-deoxy-α-D-glucopyranosyl)-myo-inositol 1-(1,2-di-O-hexadecanoyl-sn-glycerol 3-phosphate), consisting of 7 variants of either the D-myo-inositol, D-GlcpN or the phospholipid components, were prepared and tested as substrates and inhibitors of GlcNAc-PI de-N-acetylase, a genetically validated drug target enzyme responsible for the second step in the glycosylphosphatidylinositol (GPI) biosynthetic pathway of Trypanosoma brucei. The D-myo-inositol in the physiological substrate was successfully replaced by cyclohexanediol and is still a substrate for T. brucei GlcNAc-PI de-N-acetylase. However, this compound became sensitive to the stereochemistry of the glycoside linkage (the β-anomer was neither substrate or inhibitor) and the structure of the lipid moiety (the hexadecyl derivatives were inhibitors). Chemistry was successfully developed to replace the phosphate with a sulphonamide, but the compound was neither a substrate or an inhibitor, confirming the importance of the phosphate for molecular recognition. We also replaced the glucosamine by an acyclic analogue, but this also was inactive, both as a substrate and inhibitor. These findings add significantly to our understanding of substrate and inhibitor binding to the GlcNAc-PI de-N-acetylase enzyme and will have a bearing on the design of future inhibitors.
一系列 1-D-(2-氨基-2-去氧-α-D-吡喃葡萄糖基)-肌醇 1-(1,2-二-O-十六烷酰基-sn-甘油 3-磷酸)的合成类似物,由 7 种 D-肌醇、D-GlcpN 或磷脂成分的变体组成,被制备并测试为 GlcNAc-PI 去-N-乙酰基酶的底物和抑制剂,GlcNAc-PI 去-N-乙酰基酶是一种经过基因验证的药物靶标酶,负责糖基磷脂酰肌醇(GPI)生物合成途径的第二步。生理底物中的 D-肌醇成功地被环己二醇取代,仍然是 T. brucei GlcNAc-PI 去-N-乙酰基酶的底物。然而,该化合物对糖苷键的立体化学(β-异构体既不是底物也不是抑制剂)和脂质部分的结构(十六烷基衍生物是抑制剂)变得敏感。化学成功地被开发用来取代磷酸盐为磺酰胺,但该化合物既不是底物也不是抑制剂,这证实了磷酸盐对分子识别的重要性。我们还将葡萄糖胺替换为无环类似物,但它也既不是底物也不是抑制剂,这对 GlcNAc-PI 去-N-乙酰基酶的底物和抑制剂结合的理解有重要影响,并将对未来抑制剂的设计产生影响。