鞘脂病:扩大α-突触核蛋白病的范围。

IF 3.2 4区 医学 Q2 CLINICAL NEUROLOGY
Daniel Erskine, Agnieszka K Bronowska, Tiago F Outeiro, Johannes Attems
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引用次数: 0

摘要

虽然α-突触核蛋白病理通常与路易体疾病和多系统萎缩有关,但越来越多的证据表明,它也发生在一组溶酶体储存障碍中,称为鞘脂病,由一类称为鞘脂的脂质不完全降解和随后的积累引起。值得注意的是,许多引起鞘脂病的基因也是路易体疾病的风险基因,这表明这些不同疾病之间存在病因学联系。本文综述了已报道的具有α-突触核蛋白病理的神经脂质疾病:戈谢病、克拉伯病、异色性脑白质营养不良、Tay-Sachs病和Anderson-Fabry病,并描述了这些疾病的临床和病理特征,以及提示这些疾病发生α-突触核蛋白病理的证据。最后,我们评估了这些罕见疾病背后的病理机制,特别关注酶缺乏、底物积累或两者如何导致α-突触核蛋白病理的发生及其对路易体疾病的影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Sphingolipidoses: expanding the spectrum of α-synucleinopathies.

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.

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来源期刊
Journal of Neural Transmission
Journal of Neural Transmission 医学-临床神经学
CiteScore
7.20
自引率
3.00%
发文量
112
审稿时长
2 months
期刊介绍: The investigation of basic mechanisms involved in the pathogenesis of neurological and psychiatric disorders has undoubtedly deepened our knowledge of these types of disorders. The impact of basic neurosciences on the understanding of the pathophysiology of the brain will further increase due to important developments such as the emergence of more specific psychoactive compounds and new technologies. The Journal of Neural Transmission aims to establish an interface between basic sciences and clinical neurology and psychiatry. It intends to put a special emphasis on translational publications of the newest developments in the field from all disciplines of the neural sciences that relate to a better understanding and treatment of neurological and psychiatric disorders.
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