Department of Neurology, Donders Institute for Brain, Cognition, and Behavior, Radboud University Medical Center, Geert Grooteplein 10, 6525 GA, Nijmegen, The Netherlands.
Translational Metabolic Laboratory (TML), Department of Human Genetics, Radboud University Medical Center, Geert Groote Plein Zuid 10, 6525 GA, Nijmegen, The Netherlands.
Anal Bioanal Chem. 2024 Jun;416(15):3595-3604. doi: 10.1007/s00216-024-05313-w. Epub 2024 Apr 27.
Nucleotide sugars (NS) fulfil important roles in all living organisms and in humans, related defects result in severe clinical syndromes. NS can be seen as the "activated" sugars used for biosynthesis of a wide range of glycoconjugates and serve as substrates themselves for the synthesis of other nucleotide sugars. NS analysis is complicated by the presence of multiple stereoisomers without diagnostic transition ions, therefore requiring separation by liquid chromatography. In this paper, we explored weak anion-exchange/reversed-phase chromatography on a hybrid column for the separation of 17 nucleotide sugars that can occur in humans. A robust and reproducible method was established with intra- and inter-day coefficients of variation below 10% and a linear range spanning three orders of magnitude. Application to patient fibroblasts with genetic defects in mannose-1-phosphate guanylyltransferase beta, CDP-L-ribitol pyrophosphorylase A, and UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase showed abnormal levels of guanosine-5'-diphosphate-α-D-mannose (GDP-Man), cytidine-5'-diphosphate-L-ribitol (CDP-ribitol), and cytidine-5'-monophosphate-N-acetyl-β-D-neuraminic acid (CMP-Neu5Ac), respectively, in consonance with expectations based on the diagnosis. In conclusion, a novel, semi-quantitative method was established for the analysis of nucleotide sugars that can be applied to diagnose several genetic glycosylation disorders in fibroblasts and beyond.
核苷酸糖(NS)在所有生物和人类中都发挥着重要作用,相关缺陷会导致严重的临床综合征。NS 可以被视为用于合成广泛糖缀合物的“激活”糖,并本身作为合成其他核苷酸糖的底物。由于没有诊断过渡离子,因此存在多种非对映异构体,NS 分析很复杂,这需要通过液相色谱进行分离。在本文中,我们探索了混合柱上的弱阴离子交换/反相色谱法,以分离可能存在于人类中的 17 种核苷酸糖。建立了一种稳健且可重现的方法,日内和日间变异系数低于 10%,线性范围跨越三个数量级。将该方法应用于具有甘露糖-1-磷酸鸟苷酰转移酶β、CDP-L-赤藓糖醇磷酸化酶 A 和 UDP-N-乙酰葡萄糖胺 2-差向异构酶/N-乙酰-D-甘露糖胺激酶遗传缺陷的患者成纤维细胞,显示出鸟嘌呤-5'-二磷酸-α-D-甘露糖(GDP-Man)、胞苷-5'-二磷酸-L-赤藓糖醇(CDP-赤藓糖醇)和胞苷-5'-单磷酸-N-乙酰-β-D-神经氨酸(CMP-Neu5Ac)的异常水平,与基于诊断的预期相符。总之,建立了一种用于核苷酸糖分析的新型半定量方法,可用于诊断成纤维细胞和其他细胞中的几种遗传糖基化疾病。