发明名称 |
Oligonucleotide comprising an inosine for treating DMD |
摘要 |
The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD. |
申请公布号 |
AU2010239779(A1) |
申请公布日期 |
2011.11.17 |
申请号 |
AU20100239779 |
申请日期 |
2010.04.26 |
申请人 |
PROSENSA TECHNOLOGIES B.V. |
发明人 |
VAN DEUTEKOM, JUDITH CHRISTINA THEODORA;DE KIMPE, JOSEPHUS JOHANNES;PLATENBURG, GERARDUS JOHANNES |
分类号 |
C12N15/113;C12N15/11 |
主分类号 |
C12N15/113 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|