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The approach taken to treat these blood disorders with CRISPR technology in the most advanced trial doesn’t directly correct the gene variants that cause disease. It uses a clever workaround: instead of restoring healthy adult hemoglobin, the goal is to increase levels of fetal hemoglobin. This is a form of hemoglobin that fetuses make in the womb, but children and adults don’t make. We don’t know yet why humans switch from one form of hemoglobin to the other after birth, but fetal hemoglobin is not affected by the sickle cell mutation and can take the place of defective adult hemoglobin in red blood cells. This treatment can be used for both SCD and beta thalassemia.
Chart showing types of hemoglobin production over time
In SCD patients, symptoms start to show after fetal hemoglobin (HbF) levels decrease.
In individuals with SCD, symptoms start to show during infancy, after fetal hemoglobin (HbF) levels decrease. The first step of treatment is to harvest a patient’s blood stem cells directly from their blood. Next, scientists edit the genomes of these cells to turn the fetal hemoglobin gene on. Then, chemotherapy eliminates the disease-causing blood stem cells from the patient’s body. Finally, billions of genome-edited stem cells are put back into their bloodstream. These genome-edited blood stem cells are administered by IV. If it works as intended, these cells will take up residence in the bone marrow, creating a new blood stem cell population which will make edited red blood cells that produce fetal hemoglobin.
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