Gene Therapy Research: what are the goals & possible risks?

GOALS OF GENE TRANSFER THERAPY

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 proteins
Scientists are aiming to learn 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.

Many gene therapies are under investigation for a range of conditions. No gene therapies for hemophilia A or B have been determined to be safe or effective or are approved for use.

Eliminating or reducing treatment burdens

Along with identifying potential risks, ongoing research is determining whether gene therapy can help the body produce the proteins it needs, and if that could have an impact on 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 a disease. Although gene therapy may not be able to address pre-existing damage, trials are also testing whether it may be able to slow progression of any future damage.