| 发明名称 |
MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE |
| 摘要 |
The invention relates to oligonucleotides for inducing skipping of exon 53 of the dystrophin gene. The invention also relates to methods of inducing exon 53 skipping using the oligonucleotides. |
| 申请公布号 |
US2016194636(A1) |
申请公布日期 |
2016.07.07 |
| 申请号 |
US201615047233 |
申请日期 |
2016.02.18 |
| 申请人 |
Academisch Ziekenhuis Leiden h.o.d.n. LUMC |
发明人 |
Van DEUTEKOM Judith Christina Theodora;Van OMMEN Garrit-Jan Boudewijn;AARTSMA-RUS Annemieke;den DUNNEN Johannes Theodorus |
| 分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
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| 主权项 |
1. An isolated antisense oligonucleotide 15 to 80 nucleotides in length, wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA in a muscle cell, said oligonucleotide comprising a modification. |
| 地址 |
Leiden NL |
