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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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| Publicado en: | Int J Mol Sci |
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| Autores principales: | , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
MDPI
2021
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| Materias: | |
| Acceso en línea: | 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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