发明名称 |
Pharmaceutical composition for treatment of duchenne muscular dystrophy |
摘要 |
<p>The invention provides an isolated and purified DNA set forth as SEQ ID NO:15 in the Sequence Listing and an antisense oligonucleotide complementary to the DNA. The DNA represents the splicing enhancer sequence (SES) in exon 45 of human dystrophin gene, and serves as a template in preparation of the antisense oligonucleotide, which is used to induce exon 45 skipping in certain group of patient with Duchenne muscular dystrophy to restore the reading frame of dystrophin mRNA.</p> |
申请公布号 |
EP1191098(A2) |
申请公布日期 |
2002.03.27 |
申请号 |
EP20010119696 |
申请日期 |
2001.08.23 |
申请人 |
JCR PHARMACEUTICALS CO., LTD.;MATSUO, MASAFUMI |
发明人 |
MATSUO, MASAFUMI;KAMEI, SHOICHIRO |
分类号 |
A61K9/00;A61K38/00;A61K47/02;A61K47/26;A61P21/00;C07K14/47;(IPC1-7):C12N15/11;A61K31/712;A61K48/00;C07H21/00 |
主分类号 |
A61K9/00 |
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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