Therapeutic correction of hemophilia A by transplantation of hPSC-derived liver sinusoidal endothelial cell progenitors
Blair K. Gage, Simone Merlin, Cristina Olgasi, Antonia Follenzi, Gordon Keller
Abstract
mesoderm. When transplanted into immunocompromised hemophilia A mice (NSG-HA), these VECs engraft the liver, proliferate, and mature to functional LSECs that secrete bioactive FVIII capable of correcting the bleeding phenotype. Together, these findings highlight the importance of appropriate mesoderm induction for generating hPSC-derived LSECs capable of functioning in a preclinical model of hemophilia A.
Topics & Concepts
MesodermProgenitor cellInduced pluripotent stem cellBiologyCell biologyImmunologyTransplantationNODALStem cellCancer researchEmbryonic stem cellMedicineInternal medicineAnatomyGeneticsGeneRenal and related cancersPluripotent Stem Cells ResearchHemophilia Treatment and Research