U.S. Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

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The U.S. Food and Drug Administration (FDA) has approved two new groundbreaking treatments for sickle cell disease, a painful and potentially life-threatening disorder that disproportionately affects Black youth and adults. Sickle cell disease turns red blood cells into a sickle, or “C” shape, instead of the normal disc shape. This makes it hard for blood and oxygen to flow through the body, causing pain, infections, and even organ damage.

The new treatments take blood stem cells from sickle cell patients and genetically modify them in the lab using advanced technology. The modified stem cells are put back into the patient, giving them a new supply of healthy, normally shaped red blood cells that should help reduce symptoms. Scientists are hopeful that this represents an important advance in treating this life-threatening disease.

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