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Factor VIII or factor IX gene knockout rabbit, method for preparing same and use thereof

机译:因子或or因子基因基因敲除兔,其制备方法和用途

摘要

The present invention relates to a factor VIII or factor IX gene knockout rabbit, a method for preparing the same and a use thereof and, more particularly, to a transgenic rabbit whose factor VIII or factor IX gene has been knocked out through the CRISPR/Cas9 system, a method for preparing the same and a use thereof. According to the present invention, in the transgenic rabbit, whose factor VIII and/or factor IX gene has been knocked out, the functions of factor VIII and/or factor IX, which are proteins that perform critical functions for the development of hemophilia, are inhibited, such that the transgenic rabbit is useful for the development of hemophilia treatments.
机译:本发明涉及因子VIII或因子IX基因敲除的兔子,其制备方法及其用途,更具体地,涉及其因子VIII或因子IX基因已通过CRISPR / Cas9敲除的转基因兔子。系统,其制备方法及其用途。根据本发明,在已经剔除因子VIII和/或IX因子基因的转基因兔子中,因子VIII和/或IX因子的功能是对血友病发展起关键作用的蛋白质,它们是因此,转基因兔可用于发展血友病治疗。

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