Gene therapy can cure sickle cell disease for some people.

Doctors change genes in your blood-making stem cells. This lets your body make healthy red blood cells.

If it works:

• You may be cured.
• You may have less pain.
• You could stop sickle cell disease medicine (example: Hydroxyurea).
• You will probably not need more blood transfusions for sickle cell disease.
• You will likely not get new organ damage from sickle cell disease, but it will not fix any organ damage you have.
• You may not need to go to the hospital as often as before.

Gene therapy is a long, difficult treatment.

There are risks and costs to consider.

Time

• You will spend a lot of time away from your normal life.
• Treatment takes 12 months or longer, including recovery.
• You will have follow up visits for up to 15 years.

Risks

• You can have problems during and after treatment.
• Treatment is hard on your body.
• It can cause new health problems and side effects.
• It is not common, but there is a risk of cancer or death.
• It may not work for everyone.

Cost

• The financial cost is different for everyone.
• If you get gene therapy in a clinical trial, the research program will cover most of the medical costs.
• Gene therapy that is approved by the FDA can be covered by your health insurance.
• Costs for some support services may not be covered.
• There can be other costs: loss of income due to time off work, transportation, caregiver expenses, and more.

Mental Health

• Treatment can also be a mental challenge.
• You will spend a lot of time away from your normal life.
• You will miss school, work, family activities, and other events.
• You will spend more than 4 weeks in the hospital. This can make some people feel sad and alone.