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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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| Publié dans: | Int J Mol Sci |
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| Auteurs principaux: | , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
MDPI
2021
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| Sujets: | |
| Accès en ligne: | 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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