发明名称 |
MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD |
摘要 |
Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy. |
申请公布号 |
US2016002637(A1) |
申请公布日期 |
2016.01.07 |
申请号 |
US201514858416 |
申请日期 |
2015.09.18 |
申请人 |
Sarepta Therapeutics, Inc. |
发明人 |
Sazani Peter;Kole Ryszard |
分类号 |
C12N15/113 |
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 |
Cambridge MA US |