Hemophilia Research

Hemophilia is a perfect candidate for gene therapy research: the disease is caused by mutations to a single gene. Hemophilia could be cured if clinical trials discover a way to replace or “repair” the defective gene.

‘Infecting’ the Body with Gene Therapy

Hemophilia gene therapy research seeks to replace the defective gene with a normal, fully functional, gene. Researchers have been successful in developing healthy replacement genes for use in hemophilia clinical trials. These genes will be carried into the body using a vector. A vector is a method of transportation for the gene.

Gene TherapyMost vectors are harmless viruses that are able to insert the new DNA into cells. Researchers often use the adenovirus as a gene vector for hemophilia gene therapy. The virus “infects” cells with the healthy gene, overriding the mutated hemophilia gene. In clinical trials, gene vectors have been used to cure lab animals of hemophilia A and B. The body’s immune system does not appear to release antibody inhibitors when gene therapy is used.

Future gene therapy clinical trials will involve human subjects. Hemophilia researchers need to determine if the new gene (also called a viral transgene) can be passed through sperm to future generations. One potential problem is that the transgene contains extra genetic information. This extra information is required to activate the healthy gene after it “infects” cells. But it is not known how this extra genetic material will function in future generations. Hemophilia clinical trials are exploring the possibility that a patient’s cells can be infected in the laboratory and then reintroduced to the body, thereby removing the chance of extra, unwanted, genetic material being passed on to future generations.

Clinical Trials: Accessing New Hemophilia Treatments

Gene therapy research may well produce a cure for hemophilia. In order to reach this goal, clinical trials need human participants. Volunteering in clinical trials gives people with hemophilia access to world-class specialists in hemophilia treatment, as well as the chance to try new treatments long before they are available to the general public.

Hemophilia is a heredity disease: by participating in hemophilia clinical trials now, you can help find a cure for hemophilia that will benefit your children, grandchildren, and future descendants.