The Goals of Gene Transfer Therapy

Researching big ideas and their possibilities

Clinical trials are underway to determine the risks and whether there is a need for further treatment. They are also determining if the burdens of chronic disease could be reduced or eliminated for some people. It’s important to remember that the long-term effects of gene transfer therapy are also being studied and have not been determined.

Many gene therapies for hemophilia are being studied in people to determine if they are safe and effective. This educational website will not focus on any commercially available products or specific gene therapies being researched for hemophilia A or B.

Researching the Possibilities

The top three goals researchers are investigating

Introducing Working Genes

One goal of gene transfer therapy being investigated is to introduce working genes into the body to function in place of the mutated genes responsible for the genetic condition.

Producing protein

Also being researched is whether the new, working gene helps the body produce the protein it needs to function properly. For example, in hemophilia A or B, the goal is to allow the body to produce factor VIII or factor IX, respectively, on its own.

Eliminating or reducing treatment burdens

Ongoing research is trying to determine whether gene therapy can help the body produce the proteins it needs, possibly lessening or eliminating the need for other treatment and routine disease management. Research is also looking at whether it might increase or lessen the physical, mental, and emotional burdens of disease. Although gene therapy may not be able to address preexisting damage, trials are also testing whether it may be able to slow the progression of any future damage.

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