- Patent Title: Modulation of dystrophia myotonica-protein kinase (DMPK) expression
-
Application No.: US14814174Application Date: 2015-07-30
-
Publication No.: US09765338B2Publication Date: 2017-09-19
- Inventor: C. Frank Bennett , Susan M. Freier , Robert A. MacLeod , Sanjay K. Pandey , Charles A. Thornton , Thurman Wheeler , Seng H. Cheng , Andrew Leger , Bruce M. Wentworth
- Applicant: Isis Pharmaceuticals, Inc. , University Of Rochester
- Applicant Address: US CA Carlsbad
- Assignee: Ionis Pharmaceuticals, Inc.
- Current Assignee: Ionis Pharmaceuticals, Inc.
- Current Assignee Address: US CA Carlsbad
- Agency: Ionis Pharmaceuticals, Inc. Patent Dept.
- Main IPC: C12N15/11
- IPC: C12N15/11 ; 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
- US20160068845A1 MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION Public/Granted day:2016-03-10
Information query
IPC分类:
