Article
Establishment of embryonic stem cells secreting human factor VIII for cell-based treatment of hemophilia A.
Journal of thrombosis and haemostasis : JTH - 1 Aug 2008
Kasuda S, Kubo A, Sakurai Y, Irion S, Ohashi K, Tatsumi K, Nakajima Y, Saito Y, Hatake K, Pipe S W, Shima M, Yoshioka A
Abstract excerpt
BACKGROUND: Hemophilia A is an X-chromosome-linked recessive bleeding disorder resulting from an F8 gene abnormality. Although various gene therapies have been attempted with the aim of eliminating the need for factor VIII replacement therapy, obstacles to their clinical application remain. OBJECTIVES: We evaluated whether embryonic stem (ES) cells with a tetracycline-inducible system could secrete human FVIII....
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