Pharmaceutical composition for treatment of duchenne muscular dystrophy
    95.
    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

    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。

    Cells expressing a chimera receptor and their uses
    97.
    发明公开
    Cells expressing a chimera receptor and their uses 审中-公开
    Zellen,die einchimärischenRezeptor exprimieren,und deren Verwendungen

    公开(公告)号:EP0950710A2

    公开(公告)日:1999-10-20

    申请号:EP99106794.3

    申请日:1999-04-06

    摘要: The present invention provides a chimera gene as constructed by fusing the gene for the extracelluar region of human growth hormone receptor with the genes for the transmembrane and cytoplasmic regions of thrombopoietin receptor, an expression plasmid for the said gene and animal cells capable of expressing the protein encoded by the said gene.
    The human-growth-hormone dependent animal cell line transformed with an expression plasmid for the said gene are useful in screening hormones which show affinity for the chimera receptor, as well as their agonists and antagonists.

    摘要翻译: 本发明提供了通过将人生长激素受体的细胞外区域的基因与血小板生成素受体的跨膜和细胞质区域的基因融合而构建的嵌合体基因,所述基因的表达质粒和能够表达蛋白质的动物细胞 由所述基因编码。 用所述基因的表达质粒转化的人生长激素依赖性动物细胞系可用于筛选对嵌合体受体以及它们的激动剂和拮抗剂显示亲和力的激素。

    Apoptotic agents for tumor cells of the nervous system
    100.
    发明公开
    Apoptotic agents for tumor cells of the nervous system 失效
    Apoptotische MittelfürTumorzellen des Nervensystems

    公开(公告)号:EP0828005A2

    公开(公告)日:1998-03-11

    申请号:EP97115477.8

    申请日:1997-09-05

    IPC分类号: C12N15/86 A61K48/00

    摘要: According to the present invention, there are provided apoptotic agents for tumor cells of the nervous system which comprise a vector having the γ 1 34.5 gene deleted from the genome of a herpes simplex virus. The vectors are able to cause infection in tumor cells of the nervous system, thereby inducing apoptosis, and also enable virus derivatives with enhanced thymidine-kinase sensitivity to be prepared, thus opening up a way for utilizing a drug for gene therapy.

    摘要翻译: 根据本发明,提供了包含具有从单纯疱疹病毒基因组中缺失的γ134.5基因的载体的神经系统肿瘤细胞的凋亡剂。 载体能够在神经系统的肿瘤细胞中引起感染,从而诱导凋亡,并且还能够制备具有增强的胸苷激酶敏感性的病毒衍生物,从而开辟了利用药物进行基因治疗的方法。