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Intraosseous delivery of platelet-targeted factor VIII lentiviral vector in humanized NBSGW mice

We previously showed that intraosseous (IO) delivery of factor VIII (FVIII, gene F8) lentiviral vector (LV) driven by the megakaryocyte-specific promoter Gp1bα (G-F8-LV) partially corrected the bleeding phenotype in hemophilia A (HemA) mice for up to 5 months. In this study, we further characterized...

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Detalles Bibliográficos
Autores principales:	Joo, Julia H., Wang, Xuefeng, Singh, Swati, Chen, Chun-Yu, Li, Chong, Adair, Jennifer E., Kiem, Hans-Peter, Rawlings, David J., Miao, Carol H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	The American Society of Hematology 2022
Materias:	Regular Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9577625/ https://www.ncbi.nlm.nih.gov/pubmed/35849710 http://dx.doi.org/10.1182/bloodadvances.2022008079

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