发明名称 |
INHIBITING OR DOWNREGULATING GLYCOGEN SYNTHASE BY CREATING PREMATURE STOP CODONS USING ANTISENSE OLIGONUCLEOTIDES |
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. |
申请公布号 |
EP3146050(A1) |
申请公布日期 |
2017.03.29 |
申请号 |
EP20150727794 |
申请日期 |
2015.05.22 |
申请人 |
Genzyme Corporation |
发明人 |
NELSON, Carol A.;WENTWORTH, Bruce M.;SCHEULE, Ronald K.;WEEDEN, Timothy E.;CLAYTON, Nicholas P. |
分类号 |
C12N15/113;A61K31/7105;A61K31/712 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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