Article
Endothelial cells derived from patients’ induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A
12 Mar 2020
Abstract excerpt
Abstract Hemophilia A (HA) is a bleeding disorder characterized by spontaneous and prolonged hemorrhage. The disease is caused by mutations in the coagulation factor 8 gene (F8) leading to factor VIII (FVIII) deficiency. Since FVIII is primarily produced in endothelial cells (ECs) in a non-diseased human being, ECs hold great potential for development as a cell therapy for HA. We showed that HA patient-specific...
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