Medicament for treatment of Duchenne muscular dystrophy
    10.
    发明授权
    Medicament for treatment of Duchenne muscular dystrophy 有权
    杜氏肌营养不良药物

    公开(公告)号:US06653467B1

    公开(公告)日:2003-11-25

    申请号:US09667327

    申请日:2000-09-22

    IPC分类号: C07H2104

    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 the change is due to deletion of one or more nucleotides from the normal nucleotide sequence for the exons, wherein the net of the 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,其中变化是由于 从外显子的正常核苷酸序列中删除一个或多个核苷酸,其中核苷酸数量变化的净值表示为(3×N + 1)核苷酸的减少,其中N为零或自然数。