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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Udgivet i:Front Cell Dev Biol
Main Authors: Qiu, Liyan, Xie, Mi, Zhou, Miaojin, Liu, Xionghao, Hu, Zhiqing, Wu, Lingqian
Format: Artigo
Sprog:Inglês
Udgivet: Frontiers Media S.A. 2021
Fag:
Cell and Developmental Biology
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7905062/
https://ncbi.nlm.nih.gov/pubmed/33644070
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fcell.2021.630353
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