Sickle Cell Disease
- Sickle cell disease (SCD) is an inherited disease that causes anemia (low hemoglobin) and other serious problems.
- Hemoglobin is a protein in red blood cells that carries oxygen throughout the body. When a person inherits sickle cell disease, their red blood cells have abnormal hemoglobin in them which causes the cells to “sickle”.
- “Sickled” red blood cells don’t carry oxygen as effectively as normal red blood cells. They also can stick together, which can block the blood flow in small blood vessels, causing severe pain and possibly damage to vital organs – this pain is known as a vaso-occlusive crisis or a “sickle crisis.”
- People who have SCD are born with it, meaning they inherit the gene mutation from both of their parents
- People who inherit a sickle hemoglobin gene from one parent and a normal hemoglobin gene from the other parent have a condition called sickle cell trait
Currently, the only potentially curative treatment option for sickle cell disease is a bone marrow transplant (BMT, also called a stem cell transplant or allogeneic hematopoietic stem cell transplant). You can find more information about BMT using the resources below.
bluebird bio clinical studies in Sickle Cell Disease:
A global phase 1 clinical study is underway to evaluate the safety and efficacy of one-time gene therapy in people living with severe sickle cell disease. To learn more about this investigational clinical study visit clinicaltrials.gov.
The following is a representation of organizations that offer resources and information for sickle cell disease patients, their families and caregivers:
Bone Marrow Transplant Resources
Sickle Cell Disease Resources