Molecular Mechanisms and Therapeutics for Spinocerebellar Ataxia Type 2

Polina A. Egorova, Ilya B. Bezprozvanny

Research output: Contribution to journalReview articlepeer-review

7 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)1050-1073
Number of pages24
JournalNeurotherapeutics
Volume16
Issue number4
DOIs
StatePublished - Oct 1 2019

Keywords

  • Spinocerebellar ataxia type 2
  • aggregation
  • calcium signaling
  • cerebellum
  • polyglutamine disorders

ASJC Scopus subject areas

  • Pharmacology
  • Clinical Neurology
  • Pharmacology (medical)

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