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RED BLOOD CELLS EXPRESSING VON WILLEBRAND FACTOR PROTEASE AND METHODS OF USE THEREOF
RED BLOOD CELLS EXPRESSING VON WILLEBRAND FACTOR PROTEASE AND METHODS OF USE THEREOF
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机译:表达von WILLEBRAND因子蛋白酶的红色血液细胞及其使用方法
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摘要
This disclosure provides methods and compositions for treating TTP based on transfusion of a relatively small number of genetically modified red blood cells. The genetically modified red blood cells express a fusion protein including a fragment of ADAMTS13 that is enzymatically active against von Willebrand factor (VWF). The fragments of ADAMTS13 can be resistant to the inhibitors, e.g., the auto-immune antibodies, which are responsible for the acquired form of TTP.
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