Article
In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection.
Journal of thrombosis and haemostasis : JTH - 1 Aug 2014
Schroeder J A, Chen Y, Fang J, Wilcox D A, Shi Q
Abstract excerpt
BACKGROUND: Our previous studies have demonstrated that platelet-specific gene delivery to hematopoietic stem cells can induce sustained therapeutic levels of platelet factor VIII (FVIII) expression in mice with hemophilia A. OBJECTIVE: In this study, we aimed to enhance platelet FVIII expression while minimizing potential toxicities. METHODS: A novel lentiviral vector (LV), which harbors dual genes, the FVIII...
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