Boston University Amyloidosis Center, Boston University Chobanian and Avedisian School of Medicine, Boston, Massachusetts, USA.
Protein Sci. 2024 Mar;33(3):e4871. doi: 10.1002/pro.4871.
Aggregation of antibody light chain proteins is associated with the progressive disease light chain amyloidosis. Patient-derived amyloid fibrils are formed from light chain variable domain residues in non-native conformations, highlighting a requirement that light chains unfold from their native structures in order to aggregate. However, mechanistic studies of amyloid formation have primarily focused on the self-assembly of natively unstructured peptides, and the role of native state unfolding is less well understood. Using a well-studied light chain variable domain protein known as WIL, which readily aggregates in vitro under conditions where the native state predominates, we asked how the protein concentration and addition of pre-formed fibril "seeds" alter the kinetics of aggregation. Monitoring aggregation with thioflavin T fluorescence revealed a distinctly non-linear dependence on concentration, with a maximum aggregation rate observed at 8 μM protein. This behavior is consistent with formation of alternate aggregate structures in the early phases of amyloid formation. Addition of N- or C-terminal peptide tags, which did not greatly affect the folding or stability of the protein, altered the concentration dependence of aggregation. Aggregation rates increased in the presence of pre-formed seeds, but this effect did not eliminate the delay before aggregation and became saturated when the proportion of seeds added was greater than 1 in 1600. The complexity of aggregation observed in vitro highlights how multiple species may contribute to amyloid pathology in patients.
抗体轻链蛋白的聚集与进行性疾病轻链淀粉样变性有关。源自患者的淀粉样纤维由轻链可变区残基在非天然构象下形成,这突出表明轻链需要从其天然结构中展开才能聚集。然而,淀粉样形成的机制研究主要集中在天然无规构象肽的自组装上,对于天然状态展开的作用了解较少。我们使用一种研究充分的轻链可变区蛋白 WIL,在天然状态占主导的条件下,该蛋白很容易在体外聚集,我们询问了蛋白质浓度和添加预先形成的纤维“种子”如何改变聚集的动力学。用硫黄素 T 荧光监测聚集,发现浓度存在明显的非线性依赖性,在 8μM 蛋白时观察到最大聚集速率。这种行为与淀粉样形成早期形成交替聚集结构一致。添加 N 端或 C 端肽标签不会极大地影响蛋白质的折叠或稳定性,但改变了聚集的浓度依赖性。在存在预先形成的种子的情况下,聚集速率增加,但这种效应并没有消除聚集前的延迟,当添加的种子比例大于 1600 时,该效应饱和。体外观察到的聚集复杂性突出表明,多种物质可能会导致患者的淀粉样病理学。