There are two main types of hemophilia, each with varying degrees of severity. Both are X-linked recessive disorders. Hemophilia B occurs in about 1 in 25,000 males. Hemophilia causes spontaneous bleeding into organs, which can be lethal. It can also cause severe arthritis.
Current treatment consists of transfusion of clotting factors derived from human blood.
There are risks involved with transfusion of human clotting factors, such as transmission of HIV or hepatitis viruses. In other gene therapy studies where the vector was introduced into the muscle, antibodies were produced. These investigators are trying gene transfer via the liver in hopes that it will not elicit an immune response. Click on the icon to go to that experiment or result. To access the definition of a word in red, simply click on the word.