发明名称 |
ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF |
摘要 |
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. |
申请公布号 |
HRP20160225(T1) |
申请公布日期 |
2016.05.06 |
申请号 |
HR2016P000225T |
申请日期 |
2016.03.01 |
申请人 |
THE UNIVERSITY OF WESTERN AUSTRALIA |
发明人 |
Wilton, Stephen Donald;Fletcher, Sue;McClorey, Graham |
分类号 |
C12N15/113;A61K31/7088;A61K31/712;A61K48/00;C07H21/00 |
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 |
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