NOVELBIFIDOBACTERIUM BIFIDUM
    321.
    发明公开

    公开(公告)号:EP3650533A2

    公开(公告)日:2020-05-13

    申请号:EP18817528.5

    申请日:2018-06-14

    Abstract: The present invention relates to a Bifidobacterium bifidum inducing regulatory T cells (Treg), a polysaccharide derived from Bifidobacterium bifidum, and a probiotic strain producing a polysaccharide and, more particularly, a polysaccharide containing β-1-6-glucan as an effective ingredient, a probiotic strain producing β-1-6-glucan, a food comprising the polysaccharide or strain as an effective ingredient for alleviation of immune disease or inflammatory disease, a therapeutic agent comprising the polysaccharide or strain as an effective ingredient for alleviation of immune disease or inflammatory disease, a method for preparing induced regulatory T cells (iTreg) by treatment with the polysaccharide or strain, and a cell therapy product for prevention or treatment of immune disease or inflammatory disease, comprising the induced regulatory T cells prepared by the method.

    ENDONUCLEASE TARGETING BLOOD COAGULATION FACTOR VIII GENE AND COMPOSITION FOR TREATING HEMOPHILIA COMPRISING SAME
    323.
    发明公开
    ENDONUCLEASE TARGETING BLOOD COAGULATION FACTOR VIII GENE AND COMPOSITION FOR TREATING HEMOPHILIA COMPRISING SAME 审中-公开
    核酸内切酶凝血因子VIII基因和组合物,用于治疗包含相同血凝素的血友病

    公开(公告)号:EP3243529A2

    公开(公告)日:2017-11-15

    申请号:EP16735167.5

    申请日:2016-01-06

    Abstract: The present invention provides a method for inducing an inversion of normal blood coagulation factor VIII (F8) gene, a method for correcting an inversion of blood coagulation factor VIII gene in which the inversion has occurred, and a Hemophilia A patient-derived induced pluripotent stem cell in which the inversion is corrected, constructed using the same. The method of the present invention effectively reproduces the inversion of intron 1 and intron 22 of the F8 gene, which is responsible for the majority of severe hemophilia A, and thereby may be effectively used for studying the development mechanism of hemophilia A and as a research tool for screening therapeutic agents. The inversion-corrected induced pluripotent stem cell constructed according the method of the present invention enables an efficient and fundamental treatment for hemophilia A by restoring a genotype in which mutation has occurred to a wild type-like state, without limitation via normal gene or protein delivery.

    Abstract translation: 本发明提供一种诱导正常凝血因子VIII(F8)基因反转的方法,校正发生了倒位的凝血因子VIII基因的倒位的方法和血友病A患者衍生的诱导多能干 反转被修正的单元,使用它来构造。 本发明的方法有效地再现了导致大多数严重血友病A的F8基因的内含子1和内含子22的倒位,并且因此可以有效地用于研究血友病A的发展机制并且作为研究 用于筛选治疗剂的工具。 根据本发明的方法构建的反转校正的诱导性多能干细胞通过将发生突变的基因型恢复至野生型样状态而无限制地通过正常的基因或蛋白质递送而实现对血友病A的有效且基本的治疗 。

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