Creating the World’s First CRISPR Medicine, for Sickle Cell Disease

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Sickle cell disease stems from a mutation in the gene that makes hemoglobin, the protein in red blood cells that carries oxygen throughout the body. Orkin’s team and others revealed that hemoglobin has two forms — fetal and adult — and that only the adult form is affected by sickle cell mutations, while the fetal form functions normally ...


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