| 发明名称 |
RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Duchenne and Becker Muscular Dystrophy |
| 摘要 |
The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD. |
| 申请公布号 |
US2015045413(A1) |
申请公布日期 |
2015.02.12 |
| 申请号 |
US201414444244 |
申请日期 |
2014.07.28 |
| 申请人 |
Prosensa Technologies B.V. |
发明人 |
De Visser Peter Christian;Van Deutekom Judith Christina Theodora |
| 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 oligonucleotide comprising a 2′-O-methyl RNA monomer and a phosphorothioate backbone and comprising a 5-methyluracil and/or a 5-methylcytosine and/or a 2,6-diaminopurine base, said oligonucleotide being able to induce skipping of an exon of the dystrophin pre-mRNA. |
| 地址 |
Leiden NL |
