Dialing down sickle cell disease
Flipping a single molecular switch can reverse illness in an animal model of sickle cell disease, according to a study by Harvard researchers at Children’s Hospital Boston (CHB) and Dana-Farber Cancer Institute (DFCI). When a protein called BCL11A is turned off, the body is able to manufacture red blood cells with an alternate form of hemoglobin unaffected by the mutation that causes the debilitating illness. The findings were reported online today in the journal Science by a research team led by Stuart H. Orkin, Harvard Medical School’s David G. Nathan Professor of Pediatrics at the Dana-Farber/Children’s Hospital Cancer Center (DF/CHCC). The results provide strong evidence that BCL11A could be a powerful treatment target for a significant global health problem that affects between 75,000 and 100,000 people in the United States. “This study provides the first proof of principle that BCL11A might serve as a target for treating sickle cell disease and...