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

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 g...

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Detalhes bibliográficos
Main Authors: Shi, Qizhen, Fahs, Scot A., Wilcox, David A., Kuether, Erin L., Morateck, Patricia A., Mareno, Nicole, Weiler, Hartmut, Montgomery, Robert R.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2008
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2556608/
https://ncbi.nlm.nih.gov/pubmed/18495954
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2008-02-138214
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