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    Medicament for treatment of Duchenne muscular dystrophy
    1.
    发明公开
    Medicament for treatment of Duchenne muscular dystrophy 有权
    用于治疗杜氏肌营养不良的药物

    公开(公告)号：EP1544297A2

    公开(公告)日：2005-06-22

    申请号：EP05001018.0

    申请日：2000-09-27

    申请人： JCR Pharmaceuticals Co., Ltd. ,  Matsuo, Masafumi

    发明人： Matsuo, Masafumi ,  Takeshima, Yasuhiro

    IPC分类号： C12N15/11 ,  A61K31/70 ,  A61P21/00

    CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/315

    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.

    摘要翻译： 公开了包含与SEQ ID NO：1或NO：2的核苷酸序列互补的核苷酸序列的反义寡核苷酸。 反义寡核苷酸用于治疗特定类型的杜氏肌营养不良症，其归因于构成人肌营养不良蛋白mRNA中分别与外显子43或53相邻的一个或多个外显子的核苷酸数目的变化，其中所述变化归因于 从所述外显子的正常核苷酸序列中缺失一个或多个核苷酸，其中所述核苷酸数目变化的净值表示为（3×N + 1）个核苷酸的减少，其中N是零或自然数。

    Pharmaceutical composition for treatment of duchenne muscular dystrophy
    4.
    发明公开
    Pharmaceutical composition for treatment of duchenne muscular dystrophy 有权
    Pharmazutische Zusammensetzung zur Behandlung von Duchenne-Muskeldystrophie

    公开(公告)号：EP1191098A2

    公开(公告)日：2002-03-27

    申请号：EP01119696.1

    申请日：2001-08-23

    申请人： JCR PHARMACEUTICALS CO., LTD. ,  Matsuo, Masafumi

    发明人： Matsuo, Masafumi ,  Kamei, Shoichiro

    IPC分类号： C12N15/11 ,  A61P21/00 ,  A61K31/7125 ,  A61K48/00 ,  C07H21/00

    CPC分类号： C07K14/4708 ,  A61K9/0019 ,  A61K38/00 ,  A61K47/02 ,  A61K47/26

    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.

    摘要翻译： 本发明提供了序列表中SEQ ID NO：15所示的分离和纯化的DNA，以及与DNA互补的反义寡核苷酸。 DNA代表人肌营养不良蛋白基因外显子45中的剪接增强子序列（SES），并且用作制备反义寡核苷酸的模板，其用于在某些具有杜氏肌营养不良症的患者群中诱导外显子45跳跃以恢复 阅读框肌营养不良蛋白mRNA。

    Medicament for treatment of Duchenne muscular dystrophy
    5.
    发明公开
    Medicament for treatment of Duchenne muscular dystrophy 有权
    Arzneimittel zur Behandlung von Duchenne-Muskeldystrophie

    公开(公告)号：EP1160318A3

    公开(公告)日：2004-06-23

    申请号：EP00121053.3

    申请日：2000-09-27

    申请人： JCR PHARMACEUTICALS CO., LTD. ,  Matsuo, Masafumi

    发明人： Matsuo, Masafumi ,  Takeshima, Yasuhiro

    IPC分类号： C12N15/11 ,  A61K31/70 ,  A61P21/00

    CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/315

    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.

    摘要翻译： 公开了包含与SEQ ID NO：1或NO：2的核苷酸序列互补的核苷酸序列的反义寡核苷酸。 反义寡核苷酸用于治疗特定类型的Duchenne肌营养不良症，其归因于构成人肌营养不良蛋白mRNA中分别与外显子43或53相邻的一个或多个外显子的核苷酸数量的变化，其中所述改变是由于 从所述外显子的正常核苷酸序列中删除一个或多个核苷酸，其中所述核苷酸数目变化的网表示为（3×N + 1）核苷酸的减少，其中N为零或自然数。

    Medicament for treatment of Duchenne muscular dystrophy
    6.
    发明公开
    Medicament for treatment of Duchenne muscular dystrophy 有权
    “Splicing enhancer sequences”im Dystrophie Gen

    公开(公告)号：EP1160318A2

    公开(公告)日：2001-12-05

    申请号：EP00121053.3

    申请日：2000-09-27

    申请人： JCR PHARMACEUTICALS CO., LTD. ,  Matsuo, Masafumi

    发明人： Matsuo, Masafumi ,  Takeshima, Yasuhiro

    IPC分类号： C12N15/11 ,  A61K31/70 ,  A61P21/00

    CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/315

    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.

    摘要翻译： 公开了包含与SEQ ID NO：1或NO：2的核苷酸序列互补的核苷酸序列的反义寡核苷酸。 反义寡核苷酸用于治疗特定类型的Duchenne肌营养不良症，其归因于构成人肌营养不良蛋白mRNA中分别与外显子43或53相邻的一个或多个外显子的核苷酸数量的变化，其中所述改变是由于 从所述外显子的正常核苷酸序列中删除一个或多个核苷酸，其中所述核苷酸数量的所述变化的净值表示为（3×N + 1）核苷酸的减少，其中N为零或自然数。