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    • 2. 发明申请
    • ZINC FINGER NUCLEASE TARGETING THE CMAH GENE, AND A USE THEREFOR
    • ZINC指纹核心靶向CMAH基因及其用途
    • WO2012134241A3
    • 2013-01-10
    • PCT/KR2012002419
    • 2012-03-30
    • TOOLGEN INCKIM JIN SOOKIM SEOK JOONG
    • KIM JIN SOOKIM SEOK JOONG
    • C12N15/12A01K67/027C12N5/10C12N9/14C12N15/55C12N15/85
    • C12N9/16A01K67/0276A01K2227/108A01K2267/025
    • The present invention relates to: a method for making pig cells from which the swine CMAH (swine cytidine monophospho-N-acetylneuraminic acid hydroxylase) gene has been knocked out; a method for making a pig from which the swine CMAH gene has been knocked out; a method for making a pig-derived artificial organ or artificial tissue from which the swine CMAH gene has been knocked out; a zinc finger nuclease targeting the CMAH gene; a polynucleotide coding for the zinc finger nuclease; a vector comprising the polynucleotide; a method for making cells from which the CMAH gene has been knocked out by using the zinc finger nuclease; and an egg of a nonhuman animal from which the CMAH gene has been knocked out, the nonhuman animal and a kit used to make an artificial organ derived from the nonhuman animal. The present invention makes it possible to obtain a large volume of transgenic cells for creating pigs for xenogeneic organ transplantation from which the CMAH gene is eliminated, by using a zinc finger nuclease for CMAH genes.
    • 本发明涉及一种猪细胞的制造方法,其中猪CMAH(猪胞苷磷脂酰磷酸N-乙酰神经氨酸羟化酶)基因被敲除; 猪CMAH基因已被敲除的猪的制备方法; 制造猪CMAH基因已被敲除的猪源性人造器官或人造组织的方法; 靶向CMAH基因的锌指核酸酶; 编码锌指核酸酶的多核苷酸; 包含多核苷酸的载体; 通过使用锌指核酸酶来制备CMAH基因已经被敲除的细胞的方法; 和非人动物的卵,其中CMAH基因已被敲除,非人动物和用于制备来自非人动物的人造器官的试剂盒。 通过使用锌指核酸酶用于CMAH基因,本发明使得可以获得大量用于产生用于异种器官移植的猪的转基因细胞,其中CMAH基因从其中消除。