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CRISPR gene editing raises hopes of easing the pain associated with sickle cell disease

| October 17, 2019
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Victoria Gray is the first patient involved in a trial to try to treat sickle cell disease using the powerful gene-editing technique CRISPR. Image: Meredith Rizzo/NPR
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[Sickle cell] disease is caused by a genetic defect that turns red blood cells into hard, sticky, sickle-shaped cells that don’t carry oxygen well, clog the bloodstream, damage organs and cause torturous bouts of pain.

“The pain is excruciating. It’s like being in a car accident and having lightning in your chest. It’s a pain that makes a grown woman like me scream,” Gray says. “It’s an overwhelming pain.”

Like many sickle cell patients, Victoria had to drop out of school, quit work and spend weeks in the hospital away from her family. Since many sickle cell patients don’t survive past their 40s, Gray worries whether she’ll live to see her children grow up. She just turned 34.

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But Gray has hope now, because in July doctors infused billions of her own bone marrow cells back into her body, after editing them with CRISPR.

Scientists used CRISPR to modify a gene in the cells to make them produce fetal hemoglobin, a protein that babies usually stop making shortly after birth. The hope is that the protein produced through the gene-editing treatment will give sickle cell patients like Gray healthy red blood cells.

Read full, original post: A Patient Hopes Gene-Editing Can Help With Pain Of Sickle Cell Disease

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