Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A

Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A

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...

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Bibliographic Details
Main Authors: Melanie Rose, Kewa Gao, Elizabeth Cortez‐Toledo, Emmanuel Agu, Alicia A. Hyllen, Kelsey Conroy, Guangjin Pan, Jan A. Nolta, Aijun Wang, Ping Zhou
Format: Article
Language:English
Published: Wiley 2020-06-01
Series:Stem Cells Translational Medicine
Subjects:
cell therapy
endothelial cells
FVIII
gene therapy
hemophilia
induced pluripotent stem cells (iPSCs)
Online Access:https://doi.org/10.1002/sctm.19-0261

Internet

https://doi.org/10.1002/sctm.19-0261

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