Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A

Abstract excerpt

BACKGROUND: Severe hemophilia A is managed with factor VIII replacement or hemostatic products that stop or prevent bleeding. Data on gene therapy with hematopoietic stem-cell (HSC)-based expression of factor VIII for the treatment of severe hemophilia A are lacking. METHODS: ) with a myeloid-directed CD68 promoter - either without transduction enhancer (group 1) or with transduction enhancer (group 2)....

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