Gene Therapy

Gene therapy is a technique used to target a person's genetic code or DNA to treat or cure certain genetic diseases. Gene therapies work in a few different ways, depending on the gene. They can replace a damaged gene with a healthy copy of the gene, stop an overactive gene, or introduce a new or modified gene into the body to help treat disease.

There are a variety of types of gene therapy products. At Gillette, we currently use viral vector products. 

• Viral vector products: Because viruses have a natural ability to deliver genetic material into cells, some gene therapy products are transported into cells by empty viruses. Viruses used for gene therapy have been changed so they no longer cause infectious diseases. 

Side effects will be specific to the gene therapy product your provider prescribes. Ask your healthcare provider to share specific gene therapy guidelines, including expected side effects, rare but serious side effects, and the plan for monitoring any side effects following gene therapy.

Your provider will order the necessary testing before the infusion. It is important to follow pre-infusion recommendations specific to the gene therapy product that has been prescribed. Certain gene therapy products may require other medications (such as steroids) before the infusion and continue for a period of time after the infusion. Ask your healthcare provider to share specific guidelines for your particular type of gene therapy medication.

Even though the viral vectors used for gene therapy do not cause infectious disease, the patient will shed the viral vector for a few days to weeks after the gene therapy infusion. Vector shedding is when a person releases copies of a virus, usually through urine, feces, and saliva. To reduce the risk of spreading copies of the viral vector, the patient, family, and caregivers should follow proper handling of patient feces and urine. We recommend sealing disposable diapers in trash bags and then discarding them into the regular trash. Caregivers who change diapers and/or wipe children should consider wearing disposable gloves while doing so. Vigorous hand washing should be performed throughout the day, especially after coming into direct contact with patient saliva or body waste and before eating. These precautions should be followed for one month following the infusion.

Protecting caregivers and loved ones from viral shedding is important so that they do not have a reaction to the viral vector – a process called “seroconversion.” When someone is exposed to a virus, their body produces specific antibodies. This process is called seroconversion. If someone exposed to the virus develops antibodies, they may have what is called "acquired immunity." Unfortunately, people with acquired immunity would not be able to be treated with gene therapy. This is especially important for siblings or other family members with the same genetic disease. As such, we recommend that any family members with the same disease avoid direct contact with the patient who received gene therapy for 1-2 months following gene therapy infusion to lessen the risk of developing acquired immunity. 

To date, there have been no case reports of caregivers or loved ones becoming sick or ill after contact with a person who was treated with gene therapy.