2002, Number S1
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Text ExtractionA gene therapy or gene transfer approach to treatment of severe hemophilia has several attractive features. First, modest transgene expression resulting in small increases in the level of circulating F. VIII or F. IX levels would likely improve the clinical course of severe disease. The decreased bleeding observed in patients with moderate hemophilia (1–5% F. VIII or F. IX) or in those on prophylaxis regimens demonstrates this principle. Second, over-expression of the transgene product would be unlikely to result in hard to the patient. Finally, several animal models of hemophilia exist making the possibility of accurate pre-clinical experimentation a feasible enterprise.
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