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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Detalles Bibliográficos
Autores principales: Afonso-Reis, Ricardo, Afonso, Inês T., Nóbrega, Clévio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8074016/
https://www.ncbi.nlm.nih.gov/pubmed/33921915
http://dx.doi.org/10.3390/ijms22084249

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8074016/
https://www.ncbi.nlm.nih.gov/pubmed/33921915
http://dx.doi.org/10.3390/ijms22084249

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