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

Hemophilia A (HA) is caused by mutations in the coagulation factor VIII (FVIII) gene (F8). 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 ectopically expresse...

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Detalles Bibliográficos
Autores principales: Zhao, Junya, Zhou, Miaojin, Wang, Zujia, Wu, Lingqian, Hu, Zhiqing, Liang, Desheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8775870/
https://www.ncbi.nlm.nih.gov/pubmed/35054807
http://dx.doi.org/10.3390/ijms23020623