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Neurotherapeutics. 2019 Aug 21. doi: 10.1007/s13311-019-00777-6. [Epub ahead of print]

Molecular Mechanisms and Therapeutics for Spinocerebellar Ataxia Type 2.

Author information

1
Laboratory of Molecular Neurodegeneration, Peter the Great St.Petersburg Polytechnic University, St. Petersburg, 195251, Russia.
2
Laboratory of Molecular Neurodegeneration, Peter the Great St.Petersburg Polytechnic University, St. Petersburg, 195251, Russia. Ilya.Bezprozvanny@UTSouthwestern.edu.
3
Department of Physiology, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, ND12.200, Dallas, Texas, 75390, USA. Ilya.Bezprozvanny@UTSouthwestern.edu.

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.

KEYWORDS:

Spinocerebellar ataxia type 2; aggregation.; calcium signaling; cerebellum; polyglutamine disorders

PMID:
31435879
DOI:
10.1007/s13311-019-00777-6

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