Laboratory of Pathoneurochemistry, Department of Neurochemistry, Mossakowski Medical Research Institute, 02-106 Warsaw, Poland.
Int J Mol Sci. 2024 Nov 20;25(22):12448. doi: 10.3390/ijms252212448.
The abnormal deposition and intercellular propagation of disease-specific protein play a central role in the pathogenesis of many neurodegenerative disorders. Recent studies share the common observation that the formation of protein oligomers and subsequent pathological filaments is an essential step for the disease. Synucleinopathies such as Parkinson's disease (PD), dementia with Lewy bodies (DLB) or multiple system atrophy (MSA) are neurodegenerative diseases characterized by the aggregation of the α-synucleinprotein in neurons and/or in oligodendrocytes (glial cytoplasmic inclusions), neuronal loss, and astrogliosis. A similar mechanism of protein Tau-dependent neurodegeneration is a major feature of tauopathies, represented by Alzheimer's disease (AD), corticobasal degeneration (CBD), progressive supranuclear palsy (PSP), and Pick's disease (PD). The specific inhibition of the protein misfolding and their interneuronal spreading represents a promising therapeutic strategy against both disease pathology and progression. The most recent research focuses on finding potential applications targeting the pathological forms of proteins responsible for neurodegeneration. This review highlights the mechanisms relevant to protein-dependent neurodegeneration based on the most common disorders and describes current therapeutic approaches targeting protein misfolding and aggregation.
疾病特异性蛋白的异常沉积和细胞间传播在许多神经退行性疾病的发病机制中起着核心作用。最近的研究有一个共同的观察结果,即蛋白质寡聚物的形成和随后的病理性丝状体的形成是疾病的一个必要步骤。帕金森病(PD)、路易体痴呆(DLB)或多系统萎缩(MSA)等突触核蛋白病是神经退行性疾病,其特征是α-突触核蛋白在神经元和/或少突胶质细胞(神经胶质细胞质包含物)中聚集、神经元丧失和星形胶质细胞增生。蛋白质 Tau 依赖性神经退行性变的类似机制是 Tau 病的主要特征,包括阿尔茨海默病(AD)、皮质基底节变性(CBD)、进行性核上性麻痹(PSP)和匹克病(PD)。特异性抑制蛋白质错误折叠及其在神经元间的传播是针对疾病病理和进展的一种有前途的治疗策略。最近的研究重点是寻找针对导致神经退行性变的蛋白质病理性形式的潜在应用。本综述基于最常见的疾病,强调了与蛋白质依赖性神经退行性变相关的机制,并描述了针对蛋白质错误折叠和聚集的当前治疗方法。