There are different treatments that we can use to cure someone of their sickle cell disease forever. The current curative treatments we have for sickle cell disease are: myeloablative allogeneic hematopoietic stem cell transplant, non-myeloablative allogeneic hematopoietic stem cell transplant, and gene therapy.

Curative Therapies for Sickle Cell Disease

• Allogeneic Hematopoietic Stem Cell Transplant: Stem cells from a healthy person (donor) are transferred into the bloodstream of a person with a disease (recipient) to replace their own stem cells with the goal of curing their disease. This therapy requires either myeloablative or non-myeloabalative conditioning prior to the stem cell transplant.


• Gene Therapy: Unlike allogeneic hematopoietic stem cell transplant, with gene therapy the recipient is also the donor. The recipient’s own stem cells are removed and processed in the lab so that a normal gene is inserted into the stem cells to produce normal hemoglobin. The processed stem cells are transferred back to the recipient through the bloodstream. This therapy currently requires myeloablative conditioning prior to the stem cell transplant.

Treatment Related Definitions

• Donor: A person who provides stem cells to be transferred to a recipient.


• Recipient: A person with a disease who will receive healthy stem cells to cure their disease.


• Myeloablative conditioning: A pre-tranplant treatment of chemotherapy and sometimes radiation therapy used to get rid of all the cells in the bone marrow of the recipient and to suppress their immune system to allow room for stem cells from the donor to grow and to prevent the recipient from attacking the donor’s stem cells.


• Non-Myeloablative conditioning: A pre-tranplant treatment of chemotherapy and sometimes radiation therapy used to suppress the immune system to prevent the recipient from attacking the donor’s stem cells, but does not eliminate all the cells in the bone marrow.