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Researchers Correct Sickle Cell Disease in Mice

Protein therapy shows promise for eliminating symptoms of the blood disorder.

VINAY SINGH

The Burrill Report

“This discovery provides an important new target for future therapies in people with sickle cell disease”

Researchers have found a way to correct sickle cell disease in lab mice, opening the door to a potentially new way to treat the disorder in humans.

Scientists at Harvard Medical School and the University of Texas at Austin were able to correct the disease in adult mice bred to have the disorder by activating the production of fetal hemoglobin, a blood component normally produced before, but not after birth.

Using data gathered from an earlier study that identified the protein responsible for making the switch from the production of fetal hemoglobin to adult hemoglobin, the National Institutes of Health-funded team aimed to see what would happen if they blocked production of the protein in mice with sickle cell disease. They found that when they disabled the protein, the adult mice began producing fetal hemoglobin, which eliminated symptoms of the anemia.

“This discovery provides an important new target for future therapies in people with sickle cell disease,” says Susan Shurin, acting director of the NIH’s National Heart, Lung, and Blood Institute. To date, there remains no widely available cure for sickle cell disease. With further research, the principal findings of the study might be able to be tested in human trials.

Sickle cell disease results from an abnormality in hemoglobin, a protein in red blood cells that is responsible for transporting oxygen throughout the body. People with sickle cell disease have a mutation that produces sickle shaped hemoglobin instead of normally shaped adult hemoglobin. The sickle hemoglobin causes red blood cells to stiffen and become misshapen, which in turn blocks blood vessels, causes pain, and damages organs.

Sickle cell disease affects 100,000 Americans and three to five million people globally. It is most prevalent in people of African, Mediterranean, and Middle Eastern descent.



October 14, 2011
http://www.burrillreport.com/article-researchers_correct_sickle_cell_disease_in_mice.html

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