Establishment of novel cellular therapy for hemophilia A using autologous blood outgrowth endothelial progenitors

我们已经建立了体外基因转移系统，使用转基因的血液外生内皮祖细胞（BOECs），能够在较长时间内合成治疗水平的血浆FVIII。使用这种体外基因转移策略，我们可以为血友病A小鼠模型皮下植入BOECs后提供安全有效的FVIII递送。现在，我们想在血友病A狗模型中尝试这个策略。

We have established ex vivo gene transfer system using genetically-modified blood outgrowth endothelial progenitor cells(BOECs) which are capable of synthesizing therapeutic levels of plasma FVIII over a prolonged time periods.^<20> Using this ex vivo gene transfer strategy, we can provide for the safe and efficacious delivery of FVIII following subcutaneous implantation of BOECs in hemophilia A mouse model. Now, we would like to try this strategy in hemophilia A dog model.