June 5, 2015 – Special Delivery for ADAMTS13
Ectopic expression of ADAMTS13, exclusively in platelets, is able to provide effective protection in murine models of arterial thrombosis and thrombotic thrombocytopenic purpura (TTP).
TTP is a life-threatening thrombotic microangiopathy that strikes individuals with deficiency of the von Willebrand factor (VWF) cleaving protease ADAMTS13. The most common form of TTP occurs in the presence of inhibitory autoantibodies against ADAMTS13, but it can also present in families in an autosomal recessive pattern, in which affected individuals have mutations in both alleles of ADAMTS13. The absence of sufficient ADAMTS13 activity leads to the accumulation of prothrombotic ultralarge VWF multimers, which, in the presence of other triggers, initiate the formation of VWF-rich (as opposed to fibrin-rich) thrombi in the microcirculation.