Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias
Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias
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Publisher
Switzerland: MDPI AG
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Language
English
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Publisher
Switzerland: MDPI AG
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Contents
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, which translates into progressive ataxia, the main cl...
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Full title
Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8074016
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8074016
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ISSN
1422-0067,1661-6596
E-ISSN
1422-0067
DOI
10.3390/ijms22084249
