Restoration of FVIII Function and Phenotypic Rescue in Hemophilia A Mice by Transplantation of MSCs Derived From F8-Modified iPSCs

Restoration of FVIII Function and Phenotypic Rescue in Hemophilia A Mice by Transplantation of MSCs Derived From F8-Modified iPSCs

Hemophilia A (HA), an X-linked recessive congenital bleeding disorder, affects 80%–85% of patients with hemophilia. Nearly half of severe cases of hemophilia are caused by a 0.6-Mb genomic inversion (Inv22) that disrupts F8. Although viral-based gene therapy has shown therapeutic effects for hemophi...

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Bibliographic Details
Main Authors: Liyan Qiu, Mi Xie, Miaojin Zhou, Xionghao Liu, Zhiqing Hu, Lingqian Wu
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-02-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
hemophilia A
iPSCs
gene correction
ribosomal DNA
MSCs
intravenous injection
Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2021.630353/full

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