Invention Grant
- Patent Title: Compounds and methods for modulation of dystrophia myotonica-protein kinase (DMPK) expression
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Application No.: US16167783Application Date: 2018-10-23
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Publication No.: US10954519B2Publication Date: 2021-03-23
- Inventor: Eric E. Swayze , Susan M. Freier
- Applicant: Ionis Pharmaceuticals, Inc.
- Applicant Address: US CA Carlsbad
- Assignee: Ionis Pharmaceuticals, Inc.
- Current Assignee: Ionis Pharmaceuticals, Inc.
- Current Assignee Address: US CA Carlsbad
- Agency: McNeill Baur PLLC
- Main IPC: C12N15/113
- IPC: C12N15/113
Abstract:
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.
Public/Granted literature
- US20190276832A1 COMPOUNDS AND METHODS FOR MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION Public/Granted day:2019-09-12
Information query
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