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Activating fetal hemoglobin production in mice reverses sickle cell disease. |
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EurekAlert: Flipping a single molecular switch can reverse illness in a model of sickle cell disease, according to a study by researchers at Children's Hospital Boston and Dana-Farber Cancer Institute. When turned off, the switch, a protein called BCL11A, allows the body to manufacture red blood cells with an alternate form of hemoglobin unaffected by the mutation that causes the disease. The findings – reported online by a research team led by Stuart Orkin, MD, of the Dana-Farber/Children's Hospital Cancer Center (DF/CHCC) in the journal Science on October 13 – provide strong evidence that BCL11A could be a powerful treatment target for a significant global health problem, one that affects between 75,000 and 100,000 people in the United States alone.
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