- 专利标题: INHIBITING OR DOWNREGULATING GLYCOGEN SYNTHASE BY CREATING PREMATURE STOP CODONS USING ANTISENSE OLIGONUCLEOTIDES
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申请号: EP20190340.8申请日: 2015-05-22
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公开(公告)号: EP3795687A1公开(公告)日: 2021-03-24
- 发明人: NELSON, Carol A. , WENTWORTH, Bruce M. , SCHEULE, Ronald K. , WEEDEN, Timothy E. , CLAYTON, Nicholas P.
- 申请人: Genzyme Corporation
- 申请人地址: US Cambridge, MA 02142 50 Binney Street
- 代理机构: Chapman, Desmond Mark
- 优先权: US201462002294 P 20140523
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; A61K31/712 ; A61K31/7105
摘要:
The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.
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