Treatment for Hemophilia
Hemophilia B is caused by an inherited mistake in the gene for making a protein called Factor IX, which is essential for normal blood clotting. In a recent study, researchers from St. Jude’s have used adeno-associated virus (AAV) 8 as a vector to deliver the Factor IX gene along with other genetic material into the liver of 6 people afflicted with the bleeding disorder. Following treatment with the AAV 8 vector, Factor IX levels in all patients increased. Though increasing only by a few percentage points, the elevated levels of Factor IX allowed for significant improvement in symptoms of the test subjects. Even after 18 months, the patients treated with the gene therapy maintained increased levels of Factor IX, giving hope that this form of treatment is both successful and safe.