Sickle Cell Disease

The Sickle Cell Disease topic includes research on the genetics, diagnosis, and treatment of sickle cell disease, a group of inherited red blood cell disorders. It also includes research on iron overload and acute chest syndrome.

Normally, red blood cells are disc-shaped and flexible so they can move easily through the blood vessels. In sickle cell disease, red blood cells are misshaped, typically crescent- or “sickle”-shaped due to a gene mutation. Sickle-shaped red blood cells can block blood flow to the rest of the body. Sickle cell disease can be extremely painful and lead to death.

Although recent efforts have led to breakthroughs in treating and even curing the red blood cell disease, not all those with sickle cell disease can be substantially helped or cured by current treatments. Many patients, especially those in other countries, have limited access to current treatments for the disease.