Biochemistry. 2020 Nov 17;59(45):4367-4378. doi: 10.1021/acs.biochem.0c00590. Epub 2020 Nov 3.
Wild-type transthyretin-associated (ATTRwt) amyloidosis is an age-related disease that causes heart failure in older adults. This disease frequently features cardiac amyloid fibril deposits that originate from dissociation of the tetrameric protein, transthyretin (TTR). Unlike hereditary TTR (ATTRm) amyloidosis, where amino acid replacements destabilize the native protein, in ATTRwt amyloidosis, amyloid-forming TTR lacks protein sequence alterations. The initiating cause of fibril formation in ATTRwt amyloidosis is unclear, and thus, it seems plausible that other factors are involved in TTR misfolding and unregulated accumulation of wild-type TTR fibrils. We believe that clusterin (CLU, UniProtKB P10909), a plasma circulating glycoprotein, plays a role in the pathobiology of ATTRwt amyloidosis. Previously, we have suggested a role for CLU in ATTRwt amyloidosis based on our studies showing that (1) CLU codeposits with non-native TTR in amyloid fibrils from ATTRwt cardiac tissue, (2) CLU interacts only with non-native (monomeric and aggregated) forms of TTR, and (3) CLU serum levels in patients with ATTRwt are significantly lower compared to healthy controls. In the present study, we provide comprehensive detail of compositional findings from mass spectrometry analyses of amino acid and glycan content of CLU purified from ATTRwt and control sera. The characterization of oligosaccharide content in serum CLU derived from patients with ATTRwt amyloidosis is novel data. Moreover, results comparing CLU oligosaccharide variations between patient and healthy controls are original and provide further evidence for the role of CLU in ATTRwt pathobiology, possibly linked to disease-specific structural features that limit the chaperoning capacity of CLU.
野生型转甲状腺素相关(ATTRwt)淀粉样变是一种与年龄相关的疾病,可导致老年人心力衰竭。这种疾病常表现为心脏淀粉样纤维沉积物,其来源于四聚体蛋白转甲状腺素(TTR)的解离。与遗传性 TTR(ATTRm)淀粉样变不同,在遗传性 TTR 淀粉样变中,氨基酸替换会使天然蛋白不稳定,而在 ATTRwt 淀粉样变中,淀粉样形成的 TTR 缺乏蛋白序列改变。ATTRwt 淀粉样变中纤维形成的起始原因尚不清楚,因此,其他因素似乎参与了 TTR 错误折叠和野生型 TTR 纤维的不受调节积累。我们认为,血浆循环糖蛋白簇蛋白(CLU,UniProtKB P10909)在 ATTRwt 淀粉样变的病理生物学中发挥作用。以前,我们根据我们的研究表明 CLU 在 ATTRwt 淀粉样变中的作用提出了一个假设,即 (1) CLU 与 ATTRwt 心脏组织中的淀粉样纤维中非天然 TTR 共沉积,(2) CLU 仅与非天然(单体和聚集)形式的 TTR 相互作用,以及 (3) 与健康对照组相比,ATTRwt 患者的 CLU 血清水平显著降低。在本研究中,我们提供了从 ATTRwt 和对照血清中纯化的 CLU 的氨基酸和糖含量的质谱分析的组成发现的综合详细信息。从 ATTRwt 淀粉样变性患者的血清 CLU 中衍生的糖链含量的特征是新数据。此外,比较患者和健康对照组 CLU 寡糖变化的结果是原始数据,为 CLU 在 ATTRwt 病理生物学中的作用提供了进一步的证据,可能与限制 CLU 伴侣能力的疾病特异性结构特征有关。