Stem Cell Method Uses Sangamo Zinc-Finger Nucleases to Correct Sickle Cell Disease Mutation

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 United States – sickle cell disease. Published online on March 2, 2015 in the journal Blood, the study outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, that 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. The article is titled “Correction of the Sickle-Cell Disease Mutation in Human Hematopoietic Stem/Progenitor Cells.” People with sickle cell disease are born with a mutation in their beta-globin gene, which codes for part of the tetrameric protein that forms hemoglobin the protein responsible for delivering oxygen to the body through blood circulation. Typically, hemoglobin is made of two alpha chains and two beta chains. Sickle cell disease is an autosomal recessive disease, meaning that affected individuals have the sickle cell mutation in both of their beta-globin genes. The mutation causes blood stem cells – which are made in the bone marrow – to produce distorted and rigid red blood cells that resemble a crescent or “sickle” shape. 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.
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