Department of Systems Medicine, "Tor vergata" University of Rome, Via Montpellier 1, 00133, Rome, Italy.
Laboratory of Experimental Neurology, IRCCS Fondazione Santa Lucia, Rome, Italy.
Sci Rep. 2024 Nov 3;14(1):26533. doi: 10.1038/s41598-024-77859-9.
It has been shown recently, without an explanation of the possible molecular mechanisms involved, that 4-(2-hydroxyethyl)-1-piperazinepropanesulphonic (EPPS) acid effectively protects from the neurotoxicity induced by oligomers and plaques formed by the protein amyloid-β protein. Here we report the same protective effect, obtained in vitro (HT22-diff cell line) and ex vivo (hippocampal slices) models, against amyloid neurotoxicity induced by oligomers of salmon Calcitonin (sCT), which has been shown to be a good model for the study of neurodegenerative diseases. Based on biophysical studies focusing on the protein aggregation kinetic and the interaction of the aggregates with model membranes, we propose a possible molecular mechanism underlying the protective effects. Taken together, our results indicate that EPPS is able to counteract the direct association (primary aggregation) of harmless low-molecular weight aggregates (dimers and trimers) or their aggregation catalysed by surfaces present in the solution (secondary aggregation). Thus, EPPS stabilizes harmless aggregates and hinders the formation of toxic and metastable prefibrillar oligomers. Overall, our data demonstrate that EPPS is an excellent drug candidate for the treatment of neurodegeneration due to misfolded proteins, such as Alzheimer's or Parkinson's disease.
最近已经表明,尽管没有解释涉及的可能分子机制,但 4-(2-羟乙基)-1-哌嗪丙烷磺酸(EPPS)可有效保护免受由蛋白淀粉样-β蛋白形成的寡聚物和斑块引起的神经毒性。在这里,我们报告了在体外(HT22-diff 细胞系)和离体(海马切片)模型中对由鲑鱼降钙素(sCT)的寡聚物引起的淀粉样神经毒性的相同保护作用,sCT 已被证明是研究神经退行性疾病的良好模型。基于专注于蛋白聚集动力学和聚集物与模型膜相互作用的生物物理研究,我们提出了可能的分子机制。综上所述,我们的结果表明,EPPS 能够阻止无害的低分子量聚集物(二聚体和三聚体)的直接缔合(初级聚集),或阻止存在于溶液中的表面促进的聚集(次级聚集)。因此,EPPS 稳定无害的聚集物并阻碍有毒的和亚稳定的前纤维状寡聚物的形成。总体而言,我们的数据表明,EPPS 是治疗由错误折叠蛋白引起的神经变性的理想药物候选物,例如阿尔茨海默病或帕金森病。