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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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Int J Mol Sci
Egile Nagusiak: Afonso-Reis, Ricardo, Afonso, Inês T., Nóbrega, Clévio
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: MDPI 2021
Gaiak:
Sarrera elektronikoa: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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