Molecular Mechanisms and Therapeutics for Spinocerebellar Ataxia Type 2


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.

Authors Egorova PA, Bezprozvanny IB
Journal Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
Publication Date 2019 Oct;16(4):1050-1073
PubMed 31435879
PubMed Central PMC6985344
DOI 10.1007/s13311-019-00777-6

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