Bone marrow transplants and experimental gene therapy are curing the disease. If the gene therapy works long-term, sickle cell disease would be the first genetic illness ever cured by altering genes. A cure would free children and young adults from lifetimes of pain, organ damage, and the erosion of their bodies and quality of life.
The Sickle Cell Disease Association of America is dedicated to advocating for people affected by sickle cell conditions and advancing the search for a universal cure. Cohen Children’s Hospital treats more than 500 children and adolescents with sickle cell disease and is one of the largest programs in the tri-state area.
Sickle cell disease gets its name from the shape of its characteristic blood cells, resulting from a single gene mutation that deforms the hemoglobin. Instead of soft doughnuts, the red blood cells are stiff and crescent shaped. These sickled red blood cells do not carry oxygen well, if at all, and they do not flow easily through the blood vessels.