Prepare the Way for Hemophilia A Gene Therapy | NEJM - nejm.org

Congenital hemophilia A (factor VIII deficiency) is a bleeding disorder that results from pathologic variants in the gene F8 on the X chromosome. Among persons with hemophilia, those with severe disease (i.e., a factor VIII activity level of <1%) have the highest risk of spontaneous and traumatic life- and limb-threatening bleeding.¹ Poorly controlled hemophilia manifests with chronic arthropathy associated with pain, decreased mobility, and a restricted lifestyle. To correct the coagulation defect and ameliorate bleeding, the factor VIII protein or function must be restored. The standard of care for severe hemophilia A is the prevention of bleeding with regular prophylactic . . .

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