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

• Main Authors: Brennon O'Callaghan (Author), Bente Hofstra (Author), Hillary P. Handler (Author), Holly B. Kordasiewicz (Author), Tracy Cole (Author), Lisa Duvick (Author), Jillian Friedrich (Author), Orion Rainwater (Author), Praseuth Yang (Author), Michael Benneyworth (Author), Tessa Nichols-Meade (Author), Wesley Heal (Author), Rachel Ter Haar (Author), Christine Henzler (Author), Harry T. Orr (Author)
• Format: Book
• Published: Elsevier, 2020-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...