This site is an educational service brought to you by BioMarin. No gene therapies for haemophilia A or B have been approved for use or determined to be safe or effective.

What are the goals
of gene therapy research?

We typically take medicine to help us feel better, whether physically or mentally. In the case of gene therapy, clinical trials are underway to determine the risk and if, for some people, gene therapy could reduce or eliminate the need for ongoing treatment and the burdens of chronic disease. It’s important to remember that the long-term effects of gene therapy, including duration of effect, are also being studied and have not yet been determined.

SO, WHAT DO WE HOPE FOR FROM GENE THERAPY RESEARCH?

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Genes are the instructions for the body

Gene therapy aims to address specific mutations in an individual’s genetic instructions, allowing the body to produce the proteins it needs.

INTRODUCING FUNCTIONAL GENES

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One goal of gene therapy being investigated is to introduce functioning genes into the body to try to target the mutations responsible for genetic conditions.

PRODUCING PROTEIN

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Also being researched is a way for the new, functional gene to help the body produce the protein needed to function properly. For example, in haemophilia A or B, the goal is to allow the body to produce factor VIII or factor IX, respectively, on its own.

POTENTIALLY REDUCING THE NEED FOR TREATMENT

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Research is ongoing to determine if gene therapy can help the body produce the proteins it needs. This may lead to less reliance on currently available medication.

POTENTIALLY ELIMINATING OR REDUCING SYMPTOMS

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Clinical trials are evaluating whether gene therapy could eliminate or reduce routine disease management. This in turn could possibly lessen the physical, mental and emotional burden of a disease. Although gene therapy may not be able to address pre-existing damage, it may be able to mitigate progression of any existing damage.

All of these goals, as well as the risks, are currently being evaluated in clinical trials in humans.

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