Blood Disorder Breakthrough
A gene that directly affects a certain type of hemoglobin production may lead to methods of modifying the severity of blood disorders like sickle cell disease and thalassemia.
Hemoglobin is the protein in red blood cells that carries oxygen to tissues in the body. Patients with sickle cell disease have abnormal hemoglobin that sticks together causing the red blood cells to become stiff and sickle-shaped. They then can block blood vessels and rob tissues of necessary blood and oxygen. Patients with thalassemia have trouble producing adult forms of hemoglobin.
Previous studies have shown patients who continue to produce fetal hemoglobin (HbF) have much milder forms of sickle cell anemia. Now researchers at Children’s Hospital Boston and Dana-Farber Cancer Institute have found by suppressing a gene called BCL11A, HbF production improves dramatically. This finding provides new insight into the mechanisms involved in the body’s switch from producing HbF to adult hemoglobin. It could also lead to a potential new target for therapies that could dramatically alter the course of sickle cell disease and thalassemia.
There is currently one drug, called hydroxyurea, that is FDA approved to increase levels of HbF. A therapy targeting BCL11A would be the first to directly affect the natural processes involved in increasing HbF.
SOURCE: Science, published online Dec. 4, 2008
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