Scott-Manno C

Language: Spanish
References: 4
Page: 7-9
PDF size: 29.36 Kb.

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A 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.

REFERENCES

1. Herzog RW, Yang E, Couto L, et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nature Med 1999;5:56.

2. Kay M, Manno C, Ragni M, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature Genet 2000;24:257.

3. Roth DA, Tawa NE, O’Brien J, et al. Nonviral gene transfer of the gene encoding blood coagulation factor VIII in patients with severe hemophilia A. New Eng J Med 2001;344(23)1735.

4. Wang L, Nicholas T, Read M, et al. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Molec Ther 2000;1:154.