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1.发明申请TRANSGENIC ANIMALS EXPRESSING MUTANT TREX1 PROTEIN USEFUL AS A MODEL OF AUTOIMMUNE DISEASE 审中-公开表达变异TREX1蛋白的转基因动物可用作自身免疫疾病模型
公开(公告)号：WO2017123830A1
公开(公告)日：2017-07-20
申请号：PCT/US2017/013285
申请日：2017-01-13
发明人： PERRINO, Fred W.
CPC分类号： A01K67/0275 , A01K2217/072 , A01K2227/105 , A01K2267/0325
摘要： Provided herein is a recombinant or transgenic non-human mammal comprising a nucleic acid encoding a mutant three-prime exonuclease 1 (three prime repair exonuclease 1; TREX1), and in particular aspects the mammal expresses the mutant Trex1 protein. The non-human mammal is useful for identifying candidate compounds for the treatment of autoimmune disease (in human or animal, typically mammalian) subjects. Another aspect of the invention is, accordingly, a method for indentifying candidate compounds for the treatment of autoimmune disease or disorder comprising: providing the recombinant non-human mammal; administering a test substance to the recombinant non-human mammal; and determining whether said test substance reduces at least one indicia of autoimmune disease in said mammal, wherein a reduction in said at least one indicia indicates said test substance is a candidate compound for the treatment of autoimmune disease.
摘要翻译： 本文提供了包含编码突变三基因外切核酸酶1（三种主要修复外切核酸酶1; TREX1）的核酸的重组或转基因非人哺乳动物，并且在特定方面，所述哺乳动物表达突变体 Trex1蛋白。 非人哺乳动物可用于鉴定用于治疗自身免疫性疾病（在人或动物，典型地哺乳动物）受试者中的候选化合物。 因此，本发明的另一个方面是用于鉴定用于治疗自身免疫性疾病或病症的候选化合物的方法，其包括：提供重组非人类哺乳动物; 将测试物质施用于重组非人类哺乳动物; 并确定所述测试物质是否降低所述哺乳动物中至少一种自身免疫性疾病的标记，其中所述至少一种标记的减少表明所述测试物质是用于治疗自身免疫疾病的候选化合物。
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公开(公告)号：WO2022031640A1
公开(公告)日：2022-02-10
申请号：PCT/US2021/044253
申请日：2021-08-03
IPC分类号： A61K31/122 , C07C309/44 , A61P35/00
摘要： Active compounds of Formulas I – IX are provided herein that are useful as TREX1 inhibitors, which in turn is useful for stimulating the cGAS-STING pathway. As such, these compounds may be used in a method of treating disease or disorder in which modulation of TREX1 would be of benefit, such as a cancer, viral infection, or autoimmune disease.
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