Hemophilia is a disease which affects about 1 in 5000 males, case of occurrence is very rare. In United States approximately 20,000 individuals are affected by it. In fact the most popular case of it was found in the London royal family where queen Victoria herself was the carrier of this disease.
Current treatment involves taking frequent injections of concentrated factor IX protein which the patient lacks. This treatment is very expensive and must be done repeatedly for entire life.
However, researchers have come up with an alternative cure: gene therapy. Gene therapy shows possibility for permanent cure of the disease.
Scientists have packed a normal factor IX producing gene into modified virus that targets liver cells, they are the only cells capable of production of factor IX needed for clotting. The procedure aims at self-generation of clotting factor in human body itself.
Human trials have been successfully carried out with patients, with only minor side effects which are to be worked upon. In a study carried out in London with 6 patients. During the treatment period 4 out of 6 patients did not further require factor IX injections while remaining two needed it less often.
We hope that this research will eventually result in removal of the need for regular injections and significantly reduce painful bleeding and debilitating joint damage for those living with this disease, and that it can be cured.
Information source: http://www.bbc.com