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Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias

Polyglutamine spinocerebellar ataxias (PolyQ SCAs) are a group of 6 rare autosomal dominant diseases, which arise from an abnormal CAG repeat expansion in the coding region of their causative gene. These neurodegenerative ataxic disorders are characterized by progressive cerebellar degeneration, whi...

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Bibliografske podrobnosti
izdano v:Int J Mol Sci
Main Authors: Afonso-Reis, Ricardo, Afonso, Inês T., Nóbrega, Clévio
Format: Artigo
Jezik:Inglês
Izdano: MDPI 2021
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC8074016/
https://ncbi.nlm.nih.gov/pubmed/33921915
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22084249
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