| 发明名称 |
ANTISENSE-OLIGONUKLEOTIDE ZUR INDUKTION VON EXON-SKIPPING SOWIE VERFAHREN ZUR VERWENDUNG DAVON |
| 摘要 |
<p>The invention relates to antisense oligonucleotides hybridizable to target regions in exons 53, 33, 45 or 50 of the Dystrophin gene, which oligonucleotides may comprise a non-natural backbone containing non-natural sugar moieties and/or non-natural inter-nucleotide linkages, and the use of such oligonucleotides in the treatment of muscular dystrophy, e.g. Duchenne Muscular Dystrophy.</p> |
| 申请公布号 |
DE602005026386(D1) |
申请公布日期 |
2011.03.31 |
| 申请号 |
DE20056026386T |
申请日期 |
2005.06.28 |
| 申请人 |
THE UNIVERSITY OF WESTERN AUSTRALIA |
发明人 |
WILTON, STEPHEN DONALD;FLETCHER, SUE;MCCLOREY, GRAHAM |
| 分类号 |
C12N15/113;A61K31/7105;A61K31/711;A61K48/00;A61P21/00;C07H21/00 |
主分类号 |
C12N15/113 |
| 代理机构 |
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