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 AU2015231130(A1) 申请公布日期 2016.09.22
申请号 AU20150231130 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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