Posted: Thursday, March 12, 2015 7:08 PM UCLA stem-cell researchers have shown that a novel stem-cell gene therapy method could one day provide a one-time, lasting treatment for the most common inherited blood disorder in the U.S. sickle cell disease. Publishedin the journal Blood, the study outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, the gene correction method leads to the production of normal red blood cells. The study was directed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member, Dr. Donald Kohn. People with sickle cell disease are born with a mutation in their beta-globin gene, which is responsible for delivering oxygen to the body through blood circulation. The mutation causes blood stem cellswhich are made in the bone marrowto produce distorted and rigid red blood cells that resemble a crescent or sickle shape. Consequently, the abnormally shaped red blood cells do not move smoothly through blood vessels, resulting in insufficient oxygen supply to vital organs. Anyone can be born with sickle cell disease, but it occurs more frequently in African Americans and Hispanic Americans. The stem-cell … Continue reading
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