| 发明名称 |
EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY |
| 摘要 |
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described. |
| 申请公布号 |
CA2903872(A1) |
申请公布日期 |
2014.09.25 |
| 申请号 |
CA20142903872 |
申请日期 |
2014.03.14 |
| 申请人 |
SAREPTA THERAPEUTICS, INC. |
发明人 |
BESTWICK, RICHARD K.;FRANK, DIANE ELIZABETH |
| 分类号 |
C12N15/11;A61K31/7088;A61P21/00;C12N15/113 |
主分类号 |
C12N15/11 |
| 代理机构 |
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