Article
Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity.
Blood - 1 Oct 2008
Shi Qizhen, Fahs Scot A, Wilcox David A, Kuether Erin L, Morateck Patricia A, Mareno Nicole, Weiler Hartmut, Montgomery Robert R
Abstract excerpt
Although genetic induction of factor VIII (FVIII) expression in platelets can restore hemostasis in hemophilia A mice, this approach has not been studied in the clinical setting of preexisting FVIII inhibitory antibodies to determine whether such antibodies would affect therapeutic engraftment. We generated a line of transgenic mice (2bF8) that express FVIII only in platelets using the platelet-specific alphaIIb...
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