AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

在患有B型血友病的犬和鼠中，AAV介导的FIX-Padua肝脏表达可预防和清除FIX抑制剂，且不增加血栓形成倾向。