COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION

    公开(公告)号:EP3978610A2

    公开(公告)日:2022-04-06

    申请号:EP21187734.5

    申请日:2015-03-19

    IPC分类号: C12N15/113

    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.