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鞘脂贮积病:扩展α-突触核蛋白病的范围

Sphingolipidoses: expanding the spectrum of α-synucleinopathies.

作者信息

Erskine Daniel, Bronowska Agnieszka K, Outeiro Tiago F, Attems Johannes

机构信息

Metabolic Neurodegeneration Laboratory, Newcastle University, Newcastle, UK.

Translational and Clinical Research Institute, Newcastle University, Newcastle, UK.

出版信息

J Neural Transm (Vienna). 2025 Apr 17. doi: 10.1007/s00702-025-02925-z.

Abstract

Although α-synuclein pathology is typically associated with Lewy body diseases and multiple systems atrophy, increasing evidence indicates that it also occurs in a group of lysosomal storage disorders termed sphingolipidoses caused by the incomplete degradation, and subsequent accumulation, of a class of lipids termed sphingolipids. Notably, a number of genes that cause sphingolipidoses are also risk genes for Lewy body diseases, suggesting aetiological links between these distinct disorders. In the present review, we discuss the sphingolipidoses in which α-synuclein pathology has been reported: Gaucher disease, Krabbe disease, metachromatic leukodystrophy, Tay-Sachs disease and Anderson-Fabry disease, and describe the characteristic clinical and pathological features of these disorders, in addition to the evidence suggesting α-synuclein pathology occurs in these disorders. Finally, we evaluate the pathological mechanisms that underlie these rare disorders, with particular attention to how the enzymatic deficiency, substrate accumulation, or both, could contribute to the genesis of α-synuclein pathology and the implications of this for Lewy body diseases.

摘要

尽管α-突触核蛋白病理通常与路易体病和多系统萎缩相关,但越来越多的证据表明,它也出现在一类称为鞘脂贮积症的溶酶体贮积病中,这类疾病是由一类称为鞘脂的脂质不完全降解及随后的蓄积所致。值得注意的是,一些导致鞘脂贮积症的基因也是路易体病的风险基因,这表明这些不同疾病之间存在病因学联系。在本综述中,我们讨论了已报道有α-突触核蛋白病理的鞘脂贮积症:戈谢病、克拉伯病、异染性脑白质营养不良、泰-萨克斯病和安德森-法布里病,并描述了这些疾病的特征性临床和病理特征,以及提示α-突触核蛋白病理在这些疾病中出现的证据。最后,我们评估这些罕见疾病的病理机制,特别关注酶缺乏、底物蓄积或两者如何导致α-突触核蛋白病理的发生及其对路易体病的影响。

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