发明名称 |
Oligonucleotide for the treatment of muscular dystrophy patients |
摘要 |
<p>The invention relates to an oligonucleotide and to a pharmaceutical composition comprising said oligonucleotide. This oligonucleotide is able to bind to a region of a first exon from a dystrophin pre-mRNA and to a region of a second exon within the same pre-mRNA, wherein said region of said second exon has at least 50% identity with said region of said first exon, wherein said oligonucleotide is suitable for the skipping of said first and second exons of said pre-mRNA, and preferably the entire stretch of exons in between.</p> |
申请公布号 |
AU2013285698(A1) |
申请公布日期 |
2015.02.19 |
申请号 |
AU20130285698 |
申请日期 |
2013.07.03 |
申请人 |
PROSENSA TECHNOLOGIES B.V. |
发明人 |
VAN DEUTEKOM, JUDITH CHRISTINA THEODORA |
分类号 |
A61K31/7088;A61K48/00;A61P21/00;C07H21/00;C12N9/00;C12N15/113;C12Q1/68 |
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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