脊髓小脑性共济失调 2 型的分子机制与治疗。
Molecular Mechanisms and Therapeutics for Spinocerebellar Ataxia Type 2.
机构信息
Laboratory of Molecular Neurodegeneration, Peter the Great St.Petersburg Polytechnic University, St. Petersburg, 195251, Russia.
Department of Physiology, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, ND12.200, Dallas, Texas, 75390, USA.
出版信息
Neurotherapeutics. 2019 Oct;16(4):1050-1073. doi: 10.1007/s13311-019-00777-6.
Abstract
The effective therapeutic treatment and the disease-modifying therapy for spinocerebellar ataxia type 2 (SCA2) (a progressive hereditary disease caused by an expansion of polyglutamine in the ataxin-2 protein) is not available yet. At present, only symptomatic treatment and methods of palliative care are prescribed to the patients. Many attempts were made to study the physiological, molecular, and biochemical changes in SCA2 patients and in a variety of the model systems to find new therapeutic targets for SCA2 treatment. A better understanding of the uncovered molecular mechanisms of the disease allowed the scientific community to develop strategies of potential therapy and helped to create some promising therapeutic approaches for SCA2 treatment. Recent progress in this field will be discussed in this review article.
摘要
目前对于脊髓小脑性共济失调 2 型（SCA2）（一种由ataxin-2 蛋白中多聚谷氨酰胺扩展引起的进行性遗传性疾病）还没有有效的治疗方法和疾病修正治疗方法。目前，仅为患者开具对症治疗和姑息治疗方法。许多研究试图研究 SCA2 患者和各种模型系统中的生理、分子和生化变化，以寻找 SCA2 治疗的新治疗靶点。对未被发现的疾病分子机制的更好理解使科学界能够制定潜在治疗策略，并有助于为 SCA2 治疗创造一些有前途的治疗方法。本文将讨论该领域的最新进展。
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