- 专利标题: Compositions and methods for inhibiting GYS2 expression
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申请号: US16977152申请日: 2019-02-15
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公开(公告)号: US11572562B2公开(公告)日: 2023-02-07
- 发明人: Bob D. Brown , Natalie Pursell , Henryk T. Dudek , Cheng Lai
- 申请人: Dicerna Pharmaceuticals, Inc.
- 申请人地址: US MA Lexington
- 专利权人: Dicerna Pharmaceuticals, Inc.
- 当前专利权人: Dicerna Pharmaceuticals, Inc.
- 当前专利权人地址: US MA Lexington
- 代理机构: Dechert LLP
- 代理商 Andrea L. C. Reid; Gang Wang
- 国际申请: PCT/US2019/018189 WO 20190215
- 国际公布: WO2019/168687 WO 20190906
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; A61K31/7088
摘要:
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.
公开/授权文献
- US20210062197A1 COMPOSITIONS AND METHODS FOR INHIBITING GYS2 EXPRESSION 公开/授权日:2021-03-04
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