Tanz Centre for Research in Neurodegenerative Disease, University of Toronto, 60 Leonard Ave., Rm 6KD414, Tanz CRND, Krembil Discovery Tower, Toronto, ON, M5T 0S8, Canada.
Department of Neurology, Hirosaki University Graduate School of Medicine, Hirosaki, Japan.
Acta Neuropathol Commun. 2023 Nov 23;11(1):185. doi: 10.1186/s40478-023-01687-7.
Misfolded α-synuclein (α-syn) is believed to contribute to neurodegeneration in Lewy body disease (LBD) based on considerable evidence including a gene-dosage effect observed in relation to point mutations and multiplication of SNCA in familial Parkinson's disease. A contradictory concept proposes early loss of the physiological α-syn as the major driver of neurodegeneration. There is a paucity of data on SNCA transcripts in various α-syn immunoreactive cytopathologies. Here, the total cell body, nuclear, and cytoplasmic area density of SNCA transcripts in neurons without and with various α-syn immunoreactive cytopathologies in the substantia nigra and amygdala in autopsy cases of LBD (n = 5) were evaluated using RNAscope combined with immunofluorescence for disease-associated α-syn. Single-nucleus RNA sequencing was performed to elucidate cell-type specific SNCA expression in non-diseased frontal cortex (n = 3). SNCA transcripts were observed in the neuronal nucleus and cytoplasm in neurons without α-syn, those containing punctate α-syn immunoreactivity, irregular-shaped compact inclusion, and brainstem-type and cortical-type LBs. However, SNCA transcripts were only rarely found in the α-syn immunoreactive LB areas. The total cell body SNCA transcript area densities in neurons with punctate α-syn immunoreactivity were preserved but were significantly reduced in neurons with compact α-syn inclusions both in the substantia nigra and amygdala. This reduction was also observed in the cytoplasm but not in the nucleus. Only single SNCA transcripts were detected in astrocytes with or without disease-associated α-syn immunoreactivity in the amygdala. Single-nucleus RNA sequencing revealed that excitatory and inhibitory neurons, oligodendrocyte progenitor cells, oligodendrocytes, and homeostatic microglia expressed SNCA transcripts, while expression was largely absent in astrocytes and microglia. The preserved cellular SNCA expression in the more abundant non-Lewy body type α-syn cytopathologies might provide a pool for local protein production that can aggregate and serve as a seed for misfolded α-syn. Successful segregation of disease-associated α-syn is associated with the exhaustion of SNCA production in the terminal cytopathology, the Lewy body. Our observations inform therapy development focusing on targeting SNCA transcription in LBD.
错误折叠的α-突触核蛋白(α-syn)被认为是路易体病(LBD)神经退行性变的原因,这一观点有大量证据支持,包括家族性帕金森病中与点突变和 SNCA 倍增相关的基因剂量效应。相反的概念提出,生理α-syn 的早期丧失是神经退行性变的主要驱动因素。在各种α-syn 免疫反应性细胞病变中,关于 SNCA 转录本的资料很少。在这里,使用 RNAscope 结合免疫荧光法对 LBD 尸检病例的黑质和杏仁核中无和有各种α-syn 免疫反应性细胞病变的神经元中的 SNCA 转录本的总细胞体、核和细胞质面积密度进行了评估,用于与疾病相关的α-syn。对非病变额皮质(n = 3)进行了单细胞 RNA 测序以阐明特定于 SNCA 的细胞类型表达。在无α-syn 的神经元中观察到 SNCA 转录本存在于神经元核和细胞质中,那些含有点状α-syn 免疫反应性、不规则形状致密包涵体以及脑干型和皮质型 LB。然而,SNCA 转录本仅在α-syn 免疫反应性 LB 区域很少发现。在黑质和杏仁核中,点状α-syn 免疫反应性神经元中的总细胞体 SNCA 转录本面积密度保持不变,但致密α-syn 包涵体神经元中的 SNCA 转录本面积密度显著降低。这种减少也发生在细胞质中,但不在细胞核中。在杏仁核中,无论是否存在与疾病相关的α-syn 免疫反应性,星形胶质细胞中仅检测到单个 SNCA 转录本。单细胞 RNA 测序显示兴奋性和抑制性神经元、少突胶质细胞祖细胞、少突胶质细胞和稳态小胶质细胞表达 SNCA 转录本,而星形胶质细胞和小胶质细胞中表达大量缺失。在更丰富的非路易体类型α-syn 细胞病变中,保留的细胞 SNCA 表达可能为局部蛋白质产生提供一个池,该蛋白质可以聚集并作为错误折叠α-syn 的种子。与疾病相关的α-syn 的成功分离与终末细胞病变即路易体中 SNCA 产生的耗尽有关。我们的观察结果为针对 LBD 中 SNCA 转录的靶向治疗提供了信息。