Antisense Oligonucleotide Therapeutic Approach for Suppression of Ataxin-1 Expression: A Safety Assessment

Spinocerebellar ataxia type 1 (SCA1) is a lethal, autosomal dominant neurodegenerative disease caused by a polyglutamine expansion in the ATAXIN-1 (ATXN1) protein. Preclinical studies demonstrate the therapeutic efficacy of approaches that target and reduce Atxn1 expression in a non-allele-specific...

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Detalles Bibliográficos
Autores principales: O’Callaghan, Brennon, Hofstra, Bente, Handler, Hillary P., Kordasiewicz, Holly B., Cole, Tracy, Duvick, Lisa, Friedrich, Jillian, Rainwater, Orion, Yang, Praseuth, Benneyworth, Michael, Nichols-Meade, Tessa, Heal, Wesley, Ter Haar, Rachel, Henzler, Christine, Orr, Harry T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7452125/
https://www.ncbi.nlm.nih.gov/pubmed/32818920
http://dx.doi.org/10.1016/j.omtn.2020.07.030

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