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In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low MOI

BACKGROUND: Our previous studies have demonstrated that platelet-specific gene delivery to hematopoietic stem cells can induce sustained therapeutic levels of platelet-FVIII expression in hemophilia A (HA) mice. OBJECTIVE: In this study, we aimed to enhance platelet-FVIII expression while minimizing...

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Bibliografiska uppgifter
Huvudupphovsmän: Schroeder, J. A., Chen, Y., Fang, J., Wilcox, D. A., Shi, Q.
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2014
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4127102/
https://ncbi.nlm.nih.gov/pubmed/24931217
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/jth.12633
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