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8607 Roberts Drive, Suite 150 Sandy Springs, GA 30350-2237

(770) 518-8272phone    (770) 518-3310fax

8607 Roberts Drive, Suite 150 Sandy Springs, GA 30350-2237


Curing Hemophilia


ResearchAt this time there is no cure for hemophilia.  There is, however, great hope in a treatment known as gene therapy.  Several types of gene therapy are being tested.  In general, gene therapy involves taking normal genes (the ones that tell the body to make clotting factor) and putting them into the body of a person with hemophilia.  The new genes should cause the clotting factor level in the blood to rise.  People who had successful gene therapy would no longer need to take factor.  They would be cured of hemophilia.  It is possible that the genes would only provide a partial cure.  People with severe hemophilia might be raised to the level of mild hemophilia. They would need to take factor less often.

In a small number of people with hemophilia who have had liver transplants, their hemophilia has been cured.  This is because clotting factor is made in the liver.  The new livers they received made normal amounts of factor for them.  Transplants are too risky to use as a cure for hemophilia.  Liver transplants are done only to save someone's life when the liver has stopped working.