Forrest Shelley L, Kovacs Gabor G
Tanz Centre for Research in Neurodegenerative Disease, University of Toronto, Toronto, ON, Canada; Laboratory Medicine Program and Krembil Brain Institute, University Health Network, Toronto, ON, Canada; Dementia Research Centre, Macquarie Medical School, Faculty of Medicine, Health and Human Sciences, Macquarie University, Sydney, NSW, Australia.
Tanz Centre for Research in Neurodegenerative Disease, University of Toronto, Toronto, ON, Canada; Laboratory Medicine Program and Krembil Brain Institute, University Health Network, Toronto, ON, Canada; Dementia Research Centre, Macquarie Medical School, Faculty of Medicine, Health and Human Sciences, Macquarie University, Sydney, NSW, Australia; Edmond J. Safra Program in Parkinson's Disease, Rossy PSP Centre and the Morton and Gloria Shulman Movement Disorders Clinic, Toronto Western Hospital, Toronto, ON, Canada; Department of Laboratory Medicine and Pathobiology and Department of Medicine, University of Toronto, Toronto, ON, Canada.
Pathology. 2025 Mar;57(2):178-190. doi: 10.1016/j.pathol.2024.10.006. Epub 2024 Nov 14.
Neurodegenerative diseases are a pathologically, clinically and genetically diverse group of diseases characterised by selective dysfunction, loss of synaptic connectivity and neurodegeneration, and are associated with the deposition of misfolded proteins in neurons and/or glia. Molecular studies have highlighted the role of conformationally altered proteins in the pathogenesis of neurodegenerative diseases and have paved the way for developing disease-specific biomarkers that capture and differentiate the main type/s of protein abnormality responsible for neurodegenerative diseases, some of which are currently used in clinical practice. These proteins follow sequential patterns of anatomical involvement and disease spread in the brain and may also be detected in peripheral organs. Recent studies suggest that glia are likely to have an important role in pathological spread throughout the brain and even follow distinct progression patterns from neurons. In addition to morphological and molecular approaches to the classification of these disorders, a further new stratification level incorporates the structure of protein filaments detected by cryogenic electron microscopy. Rather than occurring in isolation, combined deposition of tau, amyloid-β, α-synuclein and TDP-43 are frequently observed in neurodegenerative diseases and in the ageing brain. These can be overlooked, and their clinicopathological relevance is difficult to interpret. This review provides an overview of disease pathogenesis and diagnostic implications, recent molecular and ultrastructural classification of neurodegenerative diseases, how to approach ageing-related and mixed pathologies, and the importance of the protein-based classification system for practising neuropathologists and clinicians. This review also informs general pathologists about the relevance of ongoing full body autopsy studies to understand the spectrum and pathogenesis of neurodegenerative diseases.
神经退行性疾病是一组在病理、临床和基因方面具有多样性的疾病,其特征为选择性功能障碍、突触连接丧失和神经退行性变,并且与神经元和/或神经胶质细胞中错误折叠蛋白的沉积有关。分子研究突出了构象改变的蛋白质在神经退行性疾病发病机制中的作用,并为开发疾病特异性生物标志物铺平了道路,这些生物标志物可捕捉并区分导致神经退行性疾病的主要蛋白质异常类型,其中一些目前已应用于临床实践。这些蛋白质在大脑中遵循解剖学受累和疾病传播的顺序模式,也可能在外周器官中被检测到。最近的研究表明,神经胶质细胞可能在整个大脑的病理传播中起重要作用,甚至遵循与神经元不同的进展模式。除了对这些疾病进行形态学和分子学分类的方法外,另一个新的分层水平纳入了通过低温电子显微镜检测到的蛋白质细丝结构。tau蛋白、淀粉样β蛋白、α-突触核蛋白和TDP-43的联合沉积并非孤立发生,在神经退行性疾病和衰老大脑中经常观察到。这些可能会被忽视,其临床病理相关性也难以解释。本综述概述了疾病发病机制及诊断意义、神经退行性疾病的最新分子和超微结构分类、如何处理与衰老相关的和混合性病变,以及基于蛋白质的分类系统对神经病理学家和临床医生的重要性。本综述还向普通病理学家介绍了正在进行的全身尸检研究对于理解神经退行性疾病的范围和发病机制的相关性。