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Christmas disease, also known as factor IX deficiency or hemophilia B, accounts for about 15% of all hemophilia. Patients with factor IX levels that are <1% of normal develop hemarthroses and crippling effects. Prophylaxis with intravenous infusions of clotting factor concentrate every 2 to 3 days can prevent spontaneous bleeding and improve quality of life but is inconvenient and expensive.
The gene for factor IX resides on the X chromosome and codes for a relatively small protein (55-kD) that is expressed by hepatocytes. To evaluate the safety and effectiveness of gene therapy for Christmas disease, an international team of investigators gave a single intravenous infusion of a liver-targeted adenovirus vector expressing the human factor IX…