Gene Therapy – Care at Home (1256)

What is Gene Therapy?

Your child’s disease is caused by a change in one of their genes.  Gene therapy fixes this mistake so the gene works properly.  It works by using an inactivated viral vector. 

• An inactivated viral vector is a virus that is changed in a lab.The lab puts a new copy of the abnormal gene into the virus.The lab also inactivates (kills) the virus.When the virus is killed, it will not cause an infection.
• The inactivated virus is put into your child’s body.It enters the body by injection, infusion, pill or cream.
• In your child’s body, the viral vector gives new copies of the gene to the cells.Your child’s cells will start using the new copy of the gene.
• After the new gene has been put into your child’s body, the body will work to get rid of the viral vector.This is called shedding.The way the body sheds, or gets rid of, the viral vector is through the urine (pee), stool (poop), and saliva (spit).

What do I need to do during the shedding process?

Everyone who might touch your child’s urine, stool, and saliva must wear medical gloves. They must wash their hands well with soap and water after taking the gloves off.  Hands must be washed well with soap and water after removing the gloves.

If your child wears diapers: Wear 2 pairs of medical gloves during diaper changes.  Put a pair of medical gloves on. Then put on another pair of medical gloves over the first pair. Put dirty diapers and used gloves in a plastic bag.  Then put that bag into another plastic bag before putting it in the garbage.  Wash your hands with soap and water.  Do this the whole time your child sheds the viral vector. 

Public places: Do not to use public bathrooms when possible.  Any dirty diapers and used gloves should be put in two plastic bags.  Take the plastic bags home and put in your home garbage.  Do not throw them in the public garbage.

How long will my child’s body shed the viral vector?

For the kind of gene therapy your child received, the viral vector may be shed:

in the urine for up to:   ______________________________
in the stool for up to:   ______________________________
in the saliva for up to:  ______________________________

Why is it important to do these things during the shedding process?

Being exposed to your child’s body fluids could make your body develop antibodies to the viral vector.  Antibodies are cells that attack viruses and other infections in the body.

If a person has these antibodies in the future then they cannot get gene therapy.  It is unknown how long antibodies for a viral vector last in the body.  It may be months, years, or possibly forever.

Special Information:

• Medicine called steroids may be needed during treatment. The provider will tell you if they are needed.
• Lab tests may be done every week after the gene therapy.The provider will tell you if these are needed and for how long.
• Your child might not be able to donate blood for 2 or more years after gene therapy.The team can tell you when it is safe to donate again.
• The healthcare team will wear gloves and gowns during your visits for two months after therapy.They might also wear a mask during the visit.

Reasons to call include:

• Vomiting
• Signs of upper respiratory infection
  • Cough
  • Congestion
  • Runny nose
  • Fever