发明名称 |
Medicament for treatment of Duchenne muscular dystrophy |
摘要 |
Antisense oligonucleotides comprising a nucleotide sequence complementary to the nucleotide sequence of SEQ ID NO:1 or NO:2 are disclosed. The antisense oligonucleotides are used for treatment of specific types of Duchenne muscular dystrophy which is attributed to a change in number of the nucleotides composing one or more exons adjacent to exon 43 or 53, respectively, in human dystrophin mRNA, wherein said change is due to deletion of one or more nucleotides from the normal nucleotide sequence for said exons, wherein the net of said change in number of the nucleotides is expressed as a reduction of (3xN+1) nucleotides, wherein N is zero or a natural number. |
申请公布号 |
EP1544297(A2) |
申请公布日期 |
2005.06.22 |
申请号 |
EP20050001018 |
申请日期 |
2000.09.27 |
申请人 |
JCR PHARMACEUTICALS CO., LTD.;MATSUO, MASAFUMI |
发明人 |
MATSUO, MASAFUMI;TAKESHIMA, YASUHIRO |
分类号 |
A61K38/00;A61K48/00;A61P21/00;C12N15/113 |
主分类号 |
A61K38/00 |
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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