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Flipping Gene Switch May Reverse Sickle Cell Disease

December 10, 2013; Posted by: webleed staff

Hematology researchers at The Children’s Hospital of Philadelphia (CHOP) have managed to manipulate adult blood cells to produce a form of hemoglobin that is normal absent after the newborn by flipping a gene switch.

Because fetal hemoglobin is unaffected by the genetic defect of sickle cell disease, the cell culture findings could open the door to new therapies for this blood disorder.

“Our study shows the power of a technique called forced chromatin looping in reprogramming gene expression in blood-forming cells,” said hematology researcher Jeremy W. Rupon, M.D., Ph.D., of CHOP in a press release. “If we can translate this approach to humans, we may enable new treatment options for patients.”

Rupon presented these findings on December 8th at a press conference during the annual meeting of the American Society of Hematology (ASH) in New Orleans.

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