发明名称 COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION
摘要 Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.
申请公布号 CA2942340(A1) 申请公布日期 2015.09.24
申请号 CA20152942340 申请日期 2015.03.19
申请人 IONIS PHARMACEUTICALS, INC. 发明人 FREIER, SUSAN M.;HUNG, GENE;BENNETT, C. FRANK
分类号 C12N15/113 主分类号 C12N15/113
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