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

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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Bibliographic Details
Main Authors: Brennon O’Callaghan, Bente Hofstra, Hillary P. Handler, Holly B. Kordasiewicz, Tracy Cole, Lisa Duvick, Jillian Friedrich, Orion Rainwater, Praseuth Yang, Michael Benneyworth, Tessa Nichols-Meade, Wesley Heal, Rachel Ter Haar, Christine Henzler, Harry T. Orr
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253120302195

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http://www.sciencedirect.com/science/article/pii/S2162253120302195

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