首页> 外国专利> -2- TRANSGENIC MOUSE EXPRESSING INACTIVATED HUMAN IDURONATE-2-SULPHATASE AND METHOD FOR IMPROVING A HUNTER SYNDROME TREATING AGENT USING SAME

-2- TRANSGENIC MOUSE EXPRESSING INACTIVATED HUMAN IDURONATE-2-SULPHATASE AND METHOD FOR IMPROVING A HUNTER SYNDROME TREATING AGENT USING SAME

机译:-2-转基因小鼠灭活的人类己二酸-2-硫酸酯酶和使用相同方法改善亨特综合征的治疗方法

摘要

The present invention relates to a transgenic mouse expressing an inactive human iduronate-2-sulfatase in which cysteine at amino acid position 84 is substituted with threonine. The present invention further relates to a method for improving a therapeutic agent for Hunter syndrome using the same. Specifically, the iduronate-2-sulfatase as therapeutic agent for Hunter syndrome treatment is administered to the transgenic mouse expressing the inactive human iduronate-2-sulfatase in which cysteine at amino acid position 84 was substituted with threonine, immune response to the therapeutic agent, iduronate-2-sulfatase, does not occur. Thus, the transgenic mouse can be useful for finding out immunogenicity factors except the immunogenicity of the protein component of the therapeutic agent.
机译:本发明涉及一种表达失活的人异戊二酸酯-2-硫酸酯酶的转基因小鼠,其中氨基酸位置84的半胱氨酸被苏氨酸取代。本发明还涉及一种使用该方法改善亨特氏综合症治疗剂的方法。具体而言,向表达无活性人异氰酸酯-2-硫酸酯酶的转基因小鼠施用作为用于亨特氏综合症治疗的治疗剂的异氰酸酯-2-硫酸酯酶,其中氨基酸位置84处的半胱氨酸被苏氨酸取代,对治疗剂产生免疫应答,不会发生异氰酸酯-2-硫酸酯酶。因此,转基因小鼠可用于发现免疫原性因子,除了治疗剂的蛋白质组分的免疫原性。

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