Gene Editing for Sickle Cell
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Thanks to an engineered blood cell, a Mississippi mom watched her son play football for the first time. Victoria Gray received stem cell therapy to treat her sickle cell disease. The painful blood disorder forms the hemoglobin inside red blood cells into sickle shapes.

Hemoglobin is what allows red blood cells to carry oxygen across the body. But these sickle shaped cells aren’t flexible enough to enter tiny capillaries which puts people at risk for organ damage, stroke, and death. So, what scientist did was use a relatively new technology called CRISPR to modify Victoria’s stem cells.

Rather than trying to change her defective gene, they gave her body a large dose of healthy fetal hemoglobin which our bodies make but in small amounts. First, they isolated stem cells from her blood and used CRISPR to turn on their fetal hemoglobin gene. Chemotherapy killed the rest of her defective stem cells and then doctors infused billions of the genetically edited stem cells.

A month later, the genetically modified cells began to do their job. By four months, nearly half of her total hemoglobin was the fetal version and making healthy cells. Even better, nearly ninety five percent of her red blood cells contained fetal hemoglobin. After one year, the edited cells continued to work so that she stopped needing transfusions or ER visits. Now Victoria has a chance at a normal life which gives hope to the one hundred thousand Americans with sickle cell disease.

For more information…

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