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核质转运缺陷在肌萎缩侧索硬化症中的作用。

The Role of Nucleocytoplasmic Transport Defects in Amyotrophic Lateral Sclerosis.

机构信息

Experimental Neurology, Department of Neurosciences and Leuven Brain Institute (LBI), KU Leuven-University of Leuven, B-3000 Leuven, Belgium.

Laboratory of Neurobiology, Center for Brain & Disease Research, VIB, B-3000 Leuven, Belgium.

出版信息

Int J Mol Sci. 2021 Nov 10;22(22):12175. doi: 10.3390/ijms222212175.

Abstract

There is ample evidence that nucleocytoplasmic-transport deficits could play an important role in the pathology of amyotrophic lateral sclerosis (ALS). However, the currently available data are often circumstantial and do not fully clarify the exact causal and temporal role of nucleocytoplasmic transport deficits in ALS patients. Gaining this knowledge will be of great significance in order to be able to target therapeutically nucleocytoplasmic transport and/or the proteins involved in this process. The availability of good model systems to study the nucleocytoplasmic transport process in detail will be especially crucial in investigating the effect of different mutations, as well as of other forms of stress. In this review, we discuss the evidence for the involvement of nucleocytoplasmic transport defects in ALS and the methods used to obtain these data. In addition, we provide an overview of the therapeutic strategies which could potentially counteract these defects.

摘要

有充分的证据表明,核质转运缺陷可能在肌萎缩侧索硬化症(ALS)的病理中起重要作用。然而,目前可用的数据往往是间接的,不能完全阐明核质转运缺陷在 ALS 患者中的确切因果和时间作用。获得这些知识将具有重要意义,以便能够针对核质转运和/或该过程中涉及的蛋白质进行治疗。获得良好的模型系统来详细研究核质转运过程对于研究不同突变以及其他形式的应激的影响将尤为关键。在这篇综述中,我们讨论了核质转运缺陷参与 ALS 的证据以及获得这些数据所使用的方法。此外,我们还概述了可能对抗这些缺陷的治疗策略。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f1da/8620263/023eb8c515f4/ijms-22-12175-g001.jpg

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