Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Leukemia. 2022 Aug;36(8):2076-2085. doi: 10.1038/s41375-022-01599-w. Epub 2022 May 24.
Immunoglobulin light chain (AL) amyloidosis is caused by a small, minimally proliferating B-cell/plasma-cell clone secreting a patient-unique, aggregation-prone, toxic light chain (LC). The pathogenicity of LCs is encrypted in their sequence, yet molecular determinants of amyloidogenesis are poorly understood. Higher rates of N-glycosylation among clonal κ LCs from patients with AL amyloidosis compared to other monoclonal gammopathies indicate that this post-translational modification is associated with a higher risk of developing AL amyloidosis. Here, we exploited LC sequence information from previously published amyloidogenic and control clonal LCs and from a series of 220 patients with AL amyloidosis or multiple myeloma followed at our Institutions to define sequence and spatial features of N-glycosylation, combining bioinformatics, biochemical, proteomics, structural and genetic analyses. We found peculiar sequence and spatial pattern of N-glycosylation in amyloidogenic κ LCs, with most of the N-glycosylation sites laying in the framework region 3, particularly within the E strand, and consisting mainly of the NFT sequon, setting them apart with respect to non-amyloidogenic clonal LCs. Our data further support a potential role of N-glycosylation in determining the pathogenic behavior of a subset of amyloidogenic LCs and may help refine current N-glycosylation-based prognostic assessments for patients with monoclonal gammopathies.
免疫球蛋白轻链(AL)淀粉样变性是由一个小的、增殖能力低的 B 细胞/浆细胞克隆分泌一种患者特有的、易于聚集的、有毒的轻链(LC)引起的。LC 的致病性被编码在其序列中,但淀粉样变发生的分子决定因素仍知之甚少。与其他单克隆丙种球蛋白病相比,AL 淀粉样变性患者克隆κ LC 中更高的 N-糖基化率表明,这种翻译后修饰与更高的 AL 淀粉样变性发病风险相关。在这里,我们利用先前发表的淀粉样变性和对照克隆 LC 的 LC 序列信息,以及我们机构随访的 220 例 AL 淀粉样变性或多发性骨髓瘤患者的一系列信息,来定义 N-糖基化的序列和空间特征,结合生物信息学、生化、蛋白质组学、结构和遗传分析。我们发现淀粉样变性κ LC 中存在独特的 N-糖基化序列和空间模式,大多数 N-糖基化位点位于框架区 3 中,特别是 E 链内,主要由 NFT 序列组成,与非淀粉样变性的克隆 LC 不同。我们的数据进一步支持 N-糖基化在决定一部分淀粉样变性 LC 的致病性行为方面的潜在作用,并可能有助于改进目前基于 N-糖基化的单克隆丙种球蛋白病患者的预后评估。