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Sample Essay

The evidence for Treatment of Hemophilia A and

Hemophilia B through gene therapy


Hemophilia is an inherited disorder which involves the coagulation of blood following injury. It is primarily diagnosed in male infants shortly after birth. It is approximated that the hemophilia A affects one out of every 5000 live male births worldwide while the more rare hemophilia B occurs in one out of every 25000 live male births (Dimitrios, Zaiden and Ozturk). The disease is not restricted to any specific ethnic group and can occur in any segment of society.

The disease can be life threatening if uncontrolled hemorrhage of the soft tissues or of the cranial membranes occurs. It causes debilitating pain and inflammation of the joints and muscles due to the fact that bleeding occurs frequently in load bearing joints and large muscle groups. Major as well as minor surgery in hemophiliacs can be fraught with peril unless prior treatment with coagulation factors is undertaken. Slight injuries can also be dangerous if bleeding occurs as the coagulation process is absent to prevent significant blood loss. During the last three decades there was an exponential increase in the prevalence of AIDS and hepatitis among hemophiliacs caused by improper screening of the donor blood used to produce Factor VIII and XI. Stricter controls on blood screening have led to a halt in the number of new infections but not before several million hemophiliacs succumbed to AIDS. Even with improved treatment methods, the mortality rate among hemophiliacs is still much higher than it is among the general population.

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