Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice

Our previous studies demonstrated that intraosseous (IO) infusion of lentiviral vectors (LVs) carrying a modified B domain–deleted factor VIII (FVIII) transgene driven by a megakaryocyte-specific promoter (GP1Bα promoter; G-F8/N6-LV) successfully transduced hematopoietic stem cells (HSCs) to produce...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Wang, Xuefeng, Fu, Richard Y., Li, Chong, Chen, Chun-Yu, Firrman, Jenni, Konkle, Barbara A., Zhang, Junping, Li, Lei, Xiao, Weidong, Poncz, Mortimer, Miao, Carol H.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2020
Assuntos:
Thrombosis and Hemostasis
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7686911/
https://ncbi.nlm.nih.gov/pubmed/33216891
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020002479
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