发明公开
EP1544297A3 Medicament for treatment of Duchenne muscular dystrophy
有权
Arzneitmittel zur Behandlung von Duchenne-Muskeldystrophie
- 专利标题: Medicament for treatment of Duchenne muscular dystrophy
- 专利标题(中): Arzneitmittel zur Behandlung von Duchenne-Muskeldystrophie
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申请号: EP05001018.0申请日: 2000-09-27
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公开(公告)号: EP1544297A3公开(公告)日: 2005-07-06
- 发明人: Matsuo, Masafumi , Takeshima, Yasuhiro
- 申请人: JCR Pharmaceuticals Co., Ltd. , Matsuo, Masafumi
- 申请人地址: 3-19 Kasuga-cho Ashiya-shi, 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-shi, Hyogo 659-0021 JP
- 代理机构: Behnisch, Werner, Dr.
- 优先权: JP2000125448 20000426
- 主分类号: C12N15/11
- IPC分类号: C12N15/11 ; A61K31/70 ; A61P21/00
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 (3×N+1) nucleotides, wherein N is zero or a natural number.
公开/授权文献
- EP1544297B1 Medicament for treatment of Duchenne muscular dystrophy 公开/授权日:2009-09-16
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