Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion
This paper, cited over 200 times, summarizes a phase 1 dose escalation study, 13 subjects with hemophilia A received by peripheral intravenous infusion a retroviral vector carrying a B-domain–deleted human factor VIII (hFVIII) gene. This was the first time an engineered virus was directly administered to humans. My contributions to this study were in the monitoring of patients for replication competent retrovirus and the persistence of vector gene sequences in peripheral blood mononuclear cells.
