Gene Therapy Helps Patients With Hemophilia

“During coagulation, erythrocytes become trapped in a
fibrous mesh with the help of clotting factor IX”

A recent study from the University College London and St. Jude Children’s Research Hospital reported that gene therapy cells might protect hemophilia B patients from bleeding extensively. This is truly a “landmark case” because Hemophilia is a rare inherited disorder where the blood does not undergo the coagulation process appropriately. “As a result, patients experience bleeding after injury. This type of hemorrhage can potentially damage organs and tissues”. The treatment for this disorder is called replacement therapy and this procedure must be repeated regularly “which carries risks”. The alternative method was gene therapy where the scientists conducted this study on “four patients who went through medium doses of therapy have generated enough clotting factor IX themselves that they ceased to get preventive infusions”. The “driving force” for this therapy is the virus called “AAV-8” that targeted liver cells which naturally produce clotting factor IX. Moreover, this virus does not interfere with the DNA or the cells’ normal function. So, this makes it an ideal source for the therapy. Only liver inflammation was seen in these patients, but no other side effects emerged.
Although there were previous studies that enable the scientists to think that gene therapy could be promising for correcting disorders such as hemophilia, they, in fact, produced disappointing results. According to NHLBI Director Dr. Susan, results from this study might provide “a promising step to further findings”.


http://www.nih.gov/researchmatters/december2011/12192011hemophilia.htm