Cures/Research

Gene Therapy

Gene therapy is being thoroughly researched as a cure for several genetic diseases. Out of all the genetic disorders, hemophilia has the most favorable characteristics for this potential cure. This is because hemophilia affects the bloodstream, which is easily accessible, and needs only a small rise in clotting factors to achieve a normal clotting percentage.

Gene therapy works in hemophilia by using DNA as the drug and viruses as the deliverer. A virus containing the gene that produces Factor VIII or Factor IV (in case of Hemophilia B) is injected into a large group of cells in the patient. The hope of gene therapy is to have the cell produce more of the cured cells and spread throughout the rest of the body. If successful, the patient would never need factor replacement therapy again and would be cured of hemophilia.

This diagram displays the process of gene therapy. The picture is taken from http://www.causes-of-hemophilia.com/html/hemophilia-research.php3

Currently there are several experiments, trying gene therapy on different patients. In one experiment, intramuscular injections were tested on patients of Hemophilia B. In another study, gene therapy was tested on a hemophiliac dog which proved to be temporarily successful. However, there has been no stable or certain results that determine gene therapy would work as a cure.

With time and continuous research, gene therapy may become the cure to all genetic diseases. However right now, gene therapy needs a lot of work and experimentation to prove it safe and successful enough to be considered the panacea to hemophilia or any other genetic disorder.

Sources for this page:

Hauser, Michael A. "Gene Therapy." Genetics . Ed. Richard Robinson. New York : Macmillan Reference USA , 2003. Science Resource Center . Thomson Gale. 26 November 2006
Bolton-Maggs, Paul H B, K John Pasi. "Haemophilias A and B." The Lancet Vol 361(2003): 1801-1809.
Mannuci, Pier M., Edward G.D. Tuddenham. "The Hemophilias - From Royal Genes to Gene Therapy." New England Journal of Medicine Vol. 344, No. 23(2001): 1773-1779.