Hemophilia gene therapy begins by engineering healthy genes in a laboratory and then inserting them into the genetic material of a human virus. This virus is infused into the patient and eventually cleared from the body by the liver. However, even after the virus has left the body, the healthy genes remain behind. The goal is that these engineered genes will stay in the liver cells and multiply, thereby replacing the defective genes that caused the hemophilia. As a result, the bleeding disorder will be resolved.
The infusion of the healthy genes is done as a one-time treatment, and regular follow-up is required weekly for the first year. Long-term monitoring continues for five years.