Ectopic Expression of FVIII in HPCs and MSCs Derived from hiPSCs with Site-Specific Integration of <i>ITGA2B</i> Promoter-Driven <i>BDDF8</i> Gene in Hemophilia A

Ectopic Expression of FVIII in HPCs and MSCs Derived from hiPSCs with Site-Specific Integration of <i>ITGA2B</i> Promoter-Driven <i>BDDF8</i> Gene in Hemophilia A

Hemophilia A (HA) is caused by mutations in the coagulation factor VIII (FVIII) gene <i>(F8)</i>. Gene therapy is a hopeful cure for HA; however, FVIII inhibitors formation hinders its clinical application. Given that platelets promote coagulation via locally releasing α-granule, FVIII e...

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Bibliographic Details
Main Authors: Junya Zhao, Miaojin Zhou, Zujia Wang, Lingqian Wu, Zhiqing Hu, Desheng Liang
Format: Article
Language:English
Published: MDPI AG 2022-01-01
Series:International Journal of Molecular Sciences
Subjects:
hemophilia A
platelet-targeted strategy
nonviral vector
rDNA locus
hematopoietic progenitor cell
mesenchymal stem cell
Online Access:https://www.mdpi.com/1422-0067/23/2/623

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https://www.mdpi.com/1422-0067/23/2/623

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