Invention Grant
- Patent Title: Compositions and methods for inhibiting GYS2 expression
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Application No.: US16977152Application Date: 2019-02-15
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Publication No.: US11572562B2Publication Date: 2023-02-07
- Inventor: Bob D. Brown , Natalie Pursell , Henryk T. Dudek , Cheng Lai
- Applicant: Dicerna Pharmaceuticals, Inc.
- Applicant Address: US MA Lexington
- Assignee: Dicerna Pharmaceuticals, Inc.
- Current Assignee: Dicerna Pharmaceuticals, Inc.
- Current Assignee Address: US MA Lexington
- Agency: Dechert LLP
- Agent Andrea L. C. Reid; Gang Wang
- International Application: PCT/US2019/018189 WO 20190215
- International Announcement: WO2019/168687 WO 20190906
- Main IPC: C12N15/113
- IPC: C12N15/113 ; A61K31/7088

Abstract:
This disclosure relates to oligonucleotides, compositions and methods useful for reducing GYS2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of GYS2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of GYS2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat glycogen storage diseases (e.g., GSDIa, GSDIII, GSDIV, GSDVI, and GSDIX) and related conditions.
Public/Granted literature
- US20210062197A1 COMPOSITIONS AND METHODS FOR INHIBITING GYS2 EXPRESSION Public/Granted day:2021-03-04
Information query
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