NIH/National Heart, Lung and Blood Institute
Researchers find new genetic target for sickle cell disease therapy
Published in Respiratory Therapeutics Week, December 22nd, 2008
Researchers have identified a gene that directly affects the production of a form of hemoglobin that is instrumental in modifying the severity of the inherited blood disorders sickle cell disease and thalassemia. The discovery could lead to breakthrough therapies for sickle cell disease and thalassemia, which could potentially eliminate the devastating and life-threatening complications of these diseases, such as severe pain, damage to the eyes and other organs, infections, and stroke.
"Human Fetal Hemoglobin Expression is Regulated by the Developmental Stage-Specific Repressor BCL11A," is published online in Science December 4. The study was conducted by researchers at...
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