Endothelial cells — those that line the inside of blood vessels — derived from stem cells of people with hemophilia A and modified to produce functional factor VIII were able to ease blood loss in a mouse model of the disease, a study reported.
These findings support the potential of induced pluripotent stem cells to be a patient-specific cell therapy for hemophilia A, its researchers said.
The study, “Endothelial cells derived from patients’ induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A,” was published in the journal Stem Cells Translational Medicine.
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