Wang D, Shao X, Wang Q, Pan X, Dai Y, Yao S, Yin T, Wang Z, Zhu J, Xi X, Chen Z, Chen S, Zhang G (2021) Activated factor X targeted stored in platelets as an effective gene therapy strategy for both hemophilia A and B. Clin Transl Med 11(3):e375. doi: 10.1002/ctm2.375
Summary: Treatment of hemophiliacs with inhibitors remains challenging, and new treatments are in urgent need. Coagulation factor X plays a critical role in downstream blood coagulation cascade, which could serve as a bypassing agent for hemophilia therapy. Target expression of the FXa precursor to platelets can generate a storage pool of FXa in platelet α-granules, the platelet-stored FXa is effective in treating HA and HB with inhibitors, suggesting that this could be a novel choice for hemophilia patients with inhibitors.
Usage: A single dose of CD45.2-SAP (biotinylated Anti-CD45 mixed with Streptavidin-ZAP) enabled efficient engraftment of donor cells (> 90%) and full correction of sickle-cell anemia. (3 mg/kg iv; Palchaudhuri et al.).
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