发明公开
EP1191098A2 Pharmaceutical composition for treatment of duchenne muscular dystrophy
有权
Pharmazutische Zusammensetzung zur Behandlung von Duchenne-Muskeldystrophie
- 专利标题： Pharmaceutical composition for treatment of duchenne muscular dystrophy
- 专利标题（中）： Pharmazutische Zusammensetzung zur Behandlung von Duchenne-Muskeldystrophie
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申请号： EP01119696.1申请日： 2001-08-23
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公开(公告)号： EP1191098A2公开(公告)日： 2002-03-27
- 发明人: Matsuo, Masafumi , Kamei, Shoichiro
- 申请人： JCR PHARMACEUTICALS CO., LTD. , Matsuo, Masafumi
- 申请人地址： 3-19, Kasuga-cho Ashiya, Hyogo 659-0021 JP
- 专利权人： JCR PHARMACEUTICALS CO., LTD.,Matsuo, Masafumi
- 当前专利权人： JCR PHARMACEUTICALS CO., LTD.,Matsuo, Masafumi
- 当前专利权人地址： 3-19, Kasuga-cho Ashiya, Hyogo 659-0021 JP
- 代理机构： Behnisch, Werner, Dr.
- 优先权： JP2000256547 20000825
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; A61P21/00 ; A61K31/7125 ; A61K48/00 ; C07H21/00
摘要：
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.
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