Department of Neuroscience, Institute for Translational Neuroscience, University of Minnesota, Minneapolis, USA.
Department of Neurology, Center for Neurodegeneration and Experimental Therapeutics, University of Alabama at Birmingham, Birmingham, AL, USA.
Neurotherapeutics. 2023 Jan;20(1):48-66. doi: 10.1007/s13311-023-01357-5. Epub 2023 Apr 5.
Neurodegenerative diseases are broadly characterized neuropathologically by the degeneration of vulnerable neuronal cell types in a specific brain region. The degeneration of specific cell types has informed on the various phenotypes/clinical presentations in someone suffering from these diseases. Prominent neurodegeneration of specific neurons is seen in polyglutamine expansion diseases including Huntington's disease (HD) and spinocerebellar ataxias (SCA). The clinical manifestations observed in these diseases could be as varied as the abnormalities in motor function observed in those who have Huntington's disease (HD) as demonstrated by a chorea with substantial degeneration of striatal medium spiny neurons (MSNs) or those with various forms of spinocerebellar ataxia (SCA) with an ataxic motor presentation primarily due to degeneration of cerebellar Purkinje cells. Due to the very significant nature of the degeneration of MSNs in HD and Purkinje cells in SCAs, much of the research has centered around understanding the cell autonomous mechanisms dysregulated in these neuronal cell types. However, an increasing number of studies have revealed that dysfunction in non-neuronal glial cell types contributes to the pathogenesis of these diseases. Here we explore these non-neuronal glial cell types with a focus on how each may contribute to the pathogenesis of HD and SCA and the tools used to evaluate glial cells in the context of these diseases. Understanding the regulation of supportive and harmful phenotypes of glia in disease could lead to development of novel glia-focused neurotherapeutics.
神经退行性疾病在神经病理学上的特征是特定脑区中易损神经元细胞类型的退化。特定细胞类型的退化说明了患有这些疾病的人的各种表型/临床表现。在包括亨廷顿病 (HD) 和脊髓小脑共济失调 (SCA) 在内的多聚谷氨酰胺扩展疾病中,可见到特定神经元的显著退化。这些疾病的临床表现可能多种多样,就像亨廷顿病 (HD) 患者中观察到的运动功能异常一样,表现为纹状体中型棘突神经元 (MSN) 大量退化的舞蹈症,或各种形式的脊髓小脑共济失调 (SCA) ,表现为主要由于小脑浦肯野细胞退化引起的共济失调运动。由于 HD 中的 MSN 和 SCA 中的 Purkinje 细胞退化非常严重,因此大部分研究都集中在了解这些神经元细胞类型中失调的细胞自主机制上。然而,越来越多的研究表明,非神经元神经胶质细胞类型的功能障碍也会导致这些疾病的发病机制。在这里,我们探讨了这些非神经元神经胶质细胞类型,重点介绍了它们如何导致 HD 和 SCA 的发病机制,以及用于评估这些疾病中神经胶质细胞的工具。了解胶质细胞在疾病中的支持和有害表型的调节可能会导致开发新型针对胶质细胞的神经治疗方法。