发明名称 |
COMPOUNDS AND METHODS FOR MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION |
摘要 |
Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof. |
申请公布号 |
EP3030658(A2) |
申请公布日期 |
2016.06.15 |
申请号 |
EP20140834532 |
申请日期 |
2014.08.11 |
申请人 |
IONIS PHARMACEUTICALS, INC. |
发明人 |
PANDEY, SANJAY, K.;MACLEOD, ROBERT, A.;SWAYZE, ERIC, E.;BENNETT, C., FRANK |
分类号 |
C12N15/113;A61K48/00 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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