Treatment for Blood Disease is Gene Therapy Landmark

Gene therapy hasn’t been always successful, however medical researches in Britain have had a successful gene therapy for the blood clotting disease called hemophilia B. It was done by injecting six patients that have disease with the correct form of defective gene. Hemophilia B has been around since Queen Victoria. Hemophilia B has a clotting agent known as Factor IX gene. If this gene goes bad and untreated then it can result in death. The disease occurs mostly in men since it lies on the X chromosome of which men have only one copy of and if women have it they can compensate with the other X chromosome. The trial had six patients where the doctors injected a virus into their veins. “The virus homes in on the cells of the liver, and the gene it carries then churns out correct copies of Factor IX.” (Nicholas Wade).  This would keep producing Factor IX, which would stop the blood clotting since it’s the good gene. This success had many faults and improvements over the years, but finally it worked. However, from the six patients two of them kept needing the treatment but they weren’t injected with the same virus again since the immune system was  able to attack it. I found this ironic since a virus actually helps prevent the disease from happening by giving out correct copies of the Factor IX....its actually pretty amazing how things work