| 发明名称 |
ANTISENSE OLIGOMERS AND METHODS FOR TREATING SMN-RELATED PATHOLOGIES |
| 摘要 |
An antisense oligonucleotide of 10 to 50 nucleotides comprising a targeting sequence complementary to a region near or within intron 6, intron 7, or exon 8 of the Survival Motor Neuron 2 (SMN2) gene pre-mRNA. |
| 申请公布号 |
EP3044317(A1) |
申请公布日期 |
2016.07.20 |
| 申请号 |
EP20140844805 |
申请日期 |
2014.09.12 |
| 申请人 |
THE UNIVERSITY OF WESTERN AUSTRALIA |
发明人 |
WILTON, STEPHEN DONALD;FLETCHER, SUE;PRICE, LOREN LOUISE;MITRPANT, CHALERMCHAI |
| 分类号 |
C12N15/113;A61K31/712;A61K31/7125;A61P21/00;A61P25/02;C07H21/00 |
主分类号 |
C12N15/113 |
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