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Universal RNAi Triggers for the Specific Inhibition of Mutant Huntingtin, Atrophin-1, Ataxin-3, and Ataxin-7 Expression

The expansion of CAG repeats within the coding region of associated genes is responsible for nine inherited neurodegenerative disorders including Huntington’s disease (HD), spinocerebellar ataxias (SCAs), and dentatorubral-pallidoluysian atrophy (DRPLA). Despite years of research aimed at developing...

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Detalles Bibliográficos
Autores principales: Kotowska-Zimmer, Anna, Ostrovska, Yuliya, Olejniczak, Marta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6957814/
https://www.ncbi.nlm.nih.gov/pubmed/31927329
http://dx.doi.org/10.1016/j.omtn.2019.12.012