Bhoj Lab

University of Pennsylvania

PATHOLOGY

Philadelphia · United States

NIH-funded
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NIH R01 · 2025

Selective B Cell Depletion Using Engineered T Cells As A Curative Treatment For Acquired Thrombotic Thrombocytopenic Purpura

Project Summary Immune thrombotic thrombocytopenic purpura (iTTP) is a life-threatening thrombotic microangiopathy characterized by thrombosis due to dysregulated platelet activation. iTTP results from autoantibodies directed against ADAMTS13, a plasma protease that regulates platelet activation via cleavage of von Willebrand Factor (vWF) multimers. Autoantibodies bind to and reduce ADAMTS13 activity resulting in ultralarge-vWF multimers that induce inappropriate platelet activation and microvascular thrombi leading to anemia and tissue ischemia with end-organ damage. Current therapeutic approaches oJer transient benefit and comprise daily plasma exchange therapy with or without the addition…

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