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Hemophilia gene therapy: Update

Gene transfer is an exciting and potentially important treatment approach for hemophilia A and B. Four phase I clinical trials of the safety of gene transfer in hemophilia A or B have been completed and two more trials are currently underway. The results of these trials indicate that gene transfer in hemophilia with the vectors and doses used is safe and well tolerated. Efforts continue to understand the basic biology and improve the efficiency of gene transfer.