Hemophilia A and Induced Pluripotent Stem Cells

Hemophilia A is a hemorrhage disorder inherited according to the X-linked inheritance pattern. It affects about 1 in 4,000 to 10,000 males. Permanent changes in the F8 gene result in hemophilia A. These changes result in an abnormal version of coagulation factor VIII. This abnormal condition determines the kinds of the disorder, namely, severe and mild or moderate hemophilia A. Currently, to treat hemophilia A, infusions of plasma-derived or recombinant factor VIII can be used. However, the cost is extremely high. It makes heavy burdens on the patients, their families or the health care system. Therefore, advanced therapy as iPSCs technique has attracted attention of researchers to research for curing this disorder. For example, to obtain edited DNA segments due to hemophilia A, this technique can be used. Research results in animal models have shown important advances.