Gene Therapy is Now Treating Blood Disease

Hemophilia B, also called factor IX, is a genetic disorder in which factor IX is missing and prevent clotting of the blood. There is about a 0.02% chance of a live birth being affected by the disease in the United States. Each race is affected differently by the disease and different ethnicities show different frequencies of the disease affecting live births. There are vast efforts to hopefully cure the disease on day. In Britain six patients ailed with the disease were treated for it by being injected with the correct form of the defective gene. This is quite the achievement for gene therapy, especially since it began to carry a bad reputation after the early 2000's. Many doctors say that this break through could potentially bring the field of genetic therapy back into the modern medical industry.

The general concept of gene therapy consists of replacing defective genes in any genetic disease with the corrected genetic sequence. Although it sounds simple, carrying out this task is much more difficult that thought to be. The corrected gene will be placed into a virus, with the hope that the virus will inject cells and distribute the DNA. However, the immune system seems to be a little too successful with killing viruses.

With the advance that this injection of the corrected gene of factor IX has provided however, it has not only given insight on how to treat hemophilia B, but also how to possibly treat multiple other diseases in the future. This treatment has opened many doors to possibly being able to treat other genetic disorders. Also the way that this specific blood type was treated may be able to shed some light on the ability of eukaryotes to use corrected DNA sequences. A very close family friend of ours has hemophilia, and this cure if it comes to the United States could give her her life back by reducing the number of transfusions and medications she needs to take daily. I think that the cure for this type of hemophilia is ground breaking and can only further genetic technology in the medical field.

The original article can be found here.
The National Hemophilia Foundation's link can be found here.