GlycoFi Inc., Wholly-Owned Subsidiary of Merck & Co. Inc., Lebanon, New Hampshire, United States of America.
PLoS One. 2013 May 8;8(5):e62229. doi: 10.1371/journal.pone.0062229. Print 2013.
Protein O-mannosyltransferases (PMTs) catalyze the initial reaction of protein O-mannosylation by transferring the first mannose unit onto serine and threonine residues of a nascent polypeptide being synthesized in the endoplasmic reticulum (ER). The PMTs are well conserved in eukaryotic organisms, and in vivo defects of these enzymes result in cell death in yeast and congenital diseases in humans. A group of rhodanine-3-acetic acid derivatives (PMTi) specifically inhibits PMT activity both in vitro and in vivo. As such, these chemical compounds have been effectively used to minimize the extent of O-mannosylation on heterologously produced proteins from different yeast expression hosts. However, very little is known about how these PMT-inhibitors interact with the PMT enzyme, or what structural features of the PMTs are required for inhibitor-protein interactions. To better understand the inhibitor-enzyme interactions, and to gain potential insights for developing more effective PMT-inhibitors, we isolated PMTi-resistant mutants in Pichia pastoris. In this study, we report the identification and characterization of a point mutation within the PpPMT2 gene. We demonstrate that this F664S point mutation resulted in a near complete loss of PMTi sensitivity, both in terms of growth-inhibition and reduction in O-mannosylglycan site occupancy. Our results provide genetic evidence demonstrating that the F664 residue plays a critical role in mediating the inhibitory effects of these PMTi compounds. Our data also indicate that the main target of these PMT-inhibitors in P. pastoris is Pmt2p, and that the F664 residue most likely interacts directly with the PMTi-compounds.
蛋白 O-甘露糖基转移酶(PMTs)通过将第一个甘露糖单元转移到内质网(ER)中合成的新生多肽的丝氨酸和苏氨酸残基上,催化蛋白 O-甘露糖化的初始反应。PMTs在真核生物中高度保守,这些酶的体内缺陷导致酵母细胞死亡和人类先天性疾病。一组罗丹明-3-乙酸衍生物(PMTi)特异性地抑制 PMT 在体内和体外的活性。因此,这些化学化合物已有效地用于最大限度地减少来自不同酵母表达宿主的异源产生的蛋白质的 O-甘露糖化程度。然而,人们对这些 PMT 抑制剂如何与 PMT 酶相互作用,或者 PMTs 的哪些结构特征是抑制剂-蛋白相互作用所必需的,知之甚少。为了更好地理解抑制剂-酶相互作用,并为开发更有效的 PMT 抑制剂提供潜在的见解,我们在巴斯德毕赤酵母中分离出 PMTi 抗性突变体。在这项研究中,我们报告了在 PpPMT2 基因内发现的一个点突变的鉴定和特征。我们证明,这个 F664S 点突变导致 PMTi 敏感性几乎完全丧失,无论是在生长抑制还是在 O-甘露糖基聚糖位点占有率降低方面。我们的结果提供了遗传证据,证明 F664 残基在介导这些 PMTi 化合物的抑制作用中起着关键作用。我们的数据还表明,这些 PMT 抑制剂在巴斯德毕赤酵母中的主要靶标是 Pmt2p,并且 F664 残基很可能直接与 PMTi 化合物相互作用。