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-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
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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
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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.
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