Nonviral Transfer of the Gene Encoding Coagulation Factor VIII in Patients with Severe Hemophilia A

Abstract excerpt

BACKGROUND: We tested the safety of a nonviral somatic-cell gene-therapy system in patients with severe hemophilia A. METHODS: An open-label, phase 1 trial was conducted in six patients with severe hemophilia A. Dermal fibroblasts obtained from each patient by skin biopsy were grown in culture and transfected with a plasmid containing sequences of the gene that encodes factor VIII. Cells that produced factor VIII...

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