New Developments in Hemophilia Gene Therapy

Hemophilia is an X chromosome–linked genetic bleeding disorder caused by absent or defective clotting factor, leading to spontaneous or excessive bleeding after injury.

The disease affects as many as 33,000 males in the United States.^1,2 Because of the X-linked pattern of inheritance, almost all cases occur in males, while females are mostly carriers.¹ Most people with hemophilia