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

• 主要な著者: Brennon O'Callaghan (著者), Bente Hofstra (著者), Hillary P. Handler (著者), Holly B. Kordasiewicz (著者), Tracy Cole (著者), Lisa Duvick (著者), Jillian Friedrich (著者), Orion Rainwater (著者), Praseuth Yang (著者), Michael Benneyworth (著者), Tessa Nichols-Meade (著者), Wesley Heal (著者), Rachel Ter Haar (著者), Christine Henzler (著者), Harry T. Orr (著者)
• フォーマット: 図書
• 出版事項: Elsevier, 2020-09-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: 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...