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What Is Gene Therapy? (for Parents)

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Gene therapy is an experimental treatment using genetic material to treat or prevent certain diseases. While not yet widely available, gene therapy may one day help doctors treat diseases by directly replacing the disease-causing gene.

Clinical trials are investigating gene therapy for the treatment of cancer, age-related macular degeneration and other eye diseases, certain genetic conditions and HIV/AIDS. Currently, one gene therapy medication, Luxturna®, has been approved by the U.S. Food and Drug Administration (FDA) for use in the United States. Luxturna treats certain inherited retinal (eye) diseases.

Gene therapy

How does gene therapy work?

Gene therapy works by replacing or inactivating disease-causing genes. In some cases, gene therapy introduces new genes into the body to treat a specific disease.

With gene therapy, doctors deliver a healthy copy of a gene to cells inside the body. This healthy gene may replace a damaged (mutated) gene, inactivate a mutated gene or introduce an entirely new gene.

Carriers, called vectors, transport these healthy genes into cells. In most cases, the vectors are modified viruses that do not cause disease. Vectors may also be certain types of bacteria or circular DNA molecules (plasmid DNA). Additional methods to package and deliver genetic material are also being actively investigated, such as the use of nanoparticles, encapsulating lipid molecules and the use of electric currents.

Injection or intravenous (IV) infusion introduces vectors into the body. In some cases, doctors collect cells from a patient, add vectors in a laboratory and return the vector-containing cells to the patient’s body through injection or IV infusion.

What are the risks and benefits of gene therapy?

With the exception of Luxturna which has been FDA approved, doctors are still experimenting with gene therapy. The long-term safety of such treatments has yet to be determined. Some gene therapies appear to be effective in curing certain conditions. But there is not enough evidence about gene therapy as a whole to determine all the possible risks.

Some gene therapy research indicates gene therapy may worsen symptoms or cause them to last longer. Additionally, complications of certain gene therapies may include cancer, toxicity and inflammation.

What is the outlook after gene therapy?

Your recovery depends on which medical condition gene therapy treats. Complications can be serious and can affect your outcome.

When should I ask my doctor about gene therapy?

Researchers are investigating gene therapy to treat cancer, eye diseases, some genetic conditions and HIV/AIDS. If you are interested in participating in a clinical trial involving gene therapy, speak with your doctor. Your doctor can help determine whether gene therapy might treat your condition.

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