Addictive Substance Project, Tokyo Metropolitan Institute of Medical Sciences, 2-1-6 Kamikitazawa, Setagaya-ku, Tokyo 156-8506, Japan.
PCND Neuroscience Research Institute, Poway, CA 92063 USA.
Int J Mol Sci. 2020 Apr 19;21(8):2849. doi: 10.3390/ijms21082849.
Dementia with Lewy bodies (DLB) is the second most prevalent neurodegenerative dementia after Alzheimer's disease, and is pathologically characterized by formation of intracellular inclusions called Lewy bodies, the major constituent of which is aggregated α-synuclein (αS). Currently, neither a mechanistic etiology nor an effective disease-modifying therapy for DLB has been established. Although two missense mutations of β-synuclein (βS), V70M and P123H, were identified in sporadic and familial DLB, respectively, the precise mechanisms through which βS mutations promote DLB pathogenesis remain elusive. To further clarify such mechanisms, we investigated transgenic (Tg) mice expressing P123H βS, which develop progressive neurodegeneration in the form of axonal swelling and non-motor behaviors, such as memory dysfunction and depression, which are more prominent than motor deficits. Furthermore, cross-breeding of P123H βS Tg mice with αS Tg mice worsened the neurodegenerative phenotype presumably through the pathological cross-seeding of P123H βS with αS. Collectively, we predict that βS misfolding due to gene mutations might be pathogenic. In this paper, we will discuss the possible involvement of amyloidogenic evolvability in the pathogenesis of DLB based on our previous papers regarding the P123H βS Tg mice. Given that stimulation of αS evolvability by P123H βS may underlie neuropathology in our mouse model, more radical disease-modifying therapy might be derived from the evolvability mechanism. Additionally, provided that altered βS were involved in the pathogenesis of sporadic DLB, the P123H βS Tg mice could be used for investigating the mechanism and therapy of DLB.
路易体痴呆症 (DLB) 是仅次于阿尔茨海默病的第二大常见神经退行性痴呆症,其病理学特征为细胞内包涵体的形成,称为路易体,其主要成分是聚集的α-突触核蛋白 (αS)。目前,既没有针对 DLB 的发病机制病因学,也没有有效的疾病修饰疗法。虽然β-突触核蛋白 (βS) 的两种错义突变 V70M 和 P123H 分别在散发性和家族性 DLB 中被发现,但βS 突变促进 DLB 发病机制的确切机制仍不清楚。为了进一步阐明这些机制,我们研究了表达 P123H βS 的转基因 (Tg) 小鼠,这些小鼠表现出进行性神经退行性变,表现为轴突肿胀和非运动行为,如记忆功能障碍和抑郁,这些症状比运动缺陷更为突出。此外,P123H βS Tg 小鼠与αS Tg 小鼠杂交会加重神经退行性表型,可能是通过 P123H βS 与αS 的病理性交叉播种。总之,我们预测基因突变导致的βS 错误折叠可能是致病的。在本文中,我们将根据我们之前关于 P123H βS Tg 小鼠的论文,讨论淀粉样变可进化性在 DLB 发病机制中的可能参与。鉴于 P123H βS 刺激αS 可进化性可能是我们小鼠模型神经病理学的基础,更激进的疾病修饰疗法可能源自可进化性机制。此外,如果改变的βS 参与了散发性 DLB 的发病机制,那么 P123H βS Tg 小鼠可用于研究 DLB 的发病机制和治疗方法。