20 years after gene therapy

Your experience may be different from Nia.

Doctors do not yet know what happens 5 or more years after gene therapy, because there have not been enough patients who have had this treatment yet. With the current evidence, doctors are extrapolating from the bone marrow transplant data, which hopefully will hold true.

Some people freeze eggs or sperm before bone marrow transplant.

As part of a gene therapy, you will get chemotherapy. Chemotherapy can affect your ability to have biological children in the future.

Fertility preservation might be an option for you.

You can save eggs or sperm for the future.
This is your choice. Ask your doctor about your options.
Your insurance may not pay for this. It can be very expensive.

Chemotherapy is very strong medicine.

Chemotherapy makes room in your body for the genetically-changed stem cells.

Your blood-making stem cells with the wrong instructions take up space in your bone marrow.
The genetically-changed stem cells need to be in that space.
Chemotherapy kills the stem cells with the wrong instructions, so that the genetically-changed stem cells have a place to go in your body.

Chemotherapy kills cells.

It can be used to kill cancer cells in people with cancer.
It also kills blood-making stems cells.
It kills any cells that your body makes over and over again. For example, it kills egg cells and sperm cells.

Fertility preservation options are different for everyone.

The options are different for egg cells and sperm cells.
People who have not gone through puberty have fewer options.
Even if you do fertility preservation, you still might not be able to have biological children in the future.
Your doctor can give you the latest information and explain your options.
Learn more about fertility preservation.

If you are cured, you can still give the sickle cell gene to your children.

Gene therapy does not protect your children.
Sperm and egg cells are not changed by gene therapy.
Your sperm or eggs will still carry the sickle cell gene.