发明名称 |
MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE |
摘要 |
The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA. |
申请公布号 |
CA2519863(C) |
申请公布日期 |
2015.06.23 |
申请号 |
CA20042519863 |
申请日期 |
2004.03.22 |
申请人 |
ACADEMISCH ZIEKENHUIS LEIDEN |
发明人 |
VAN OMMEN, GARRIT-JAN BOUDEWIJN;VAN DEUTEKOM, JUDITH CHRISTINA THEODORA;DEN DUNNEN, JOHANNES THEODORUS;AARTSMA-RUS, ANNEMIEKE |
分类号 |
A61K31/7088;A61K38/00;A61K48/00;C12N15/113 |
主分类号 |
A61K31/7088 |
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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