Health Conditions

What is on the horizon?

A major focus of research in hemophilia centers on gene therapy. Because only one defective gene is involved, replacing that gene effectively will cure hemophilia. In multiple studies, normal clotting factor genes have been introduced in laboratory animals such as mice and dogs that had hemophilia. In some of the studies, increased production of clotting factors has lasted for more than a year. Only small amounts of clotting factor production was seen in some of the test animals, however, and many developed antibodies that limited clotting factor production. Genetic studies in humans with hemophilia are not yet feasible. Although gene therapy may eventually replace treatment for individuals with hemophilia, it will not eliminate defective genes in carriers, who could still pass hemophilia to their children. Currently studies are being conducted for type B hemophilia to determine the safety of the gene transfers.

A less well-studied, but potentially more useful way of producing clotting factor products for treating hemophilia may be breeding herds of ?transgenic? animals. These animals, such as goats or pigs, have been modified genetically to produce human clotting factors in their milk. Drinking milk fortified with clotting factors may supplement or even replace injections of clotting factors, as well as help to prevent inhibitors. It would also extend treatment options for individuals with hemophilia who live in remote areas of the world.