. Patients with sickle cell inherit a mutation in the gene responsible for producing hemoglobin, which is the protein that carries oxygen in red blood cells. The condition results in the production of abnormal hemoglobin, which causes normal spherical red blood cells to become crescent-shaped, which prevents their ability to flow smoothly through the bloodstream. Consequently, patients with the disease can experience severe pain, organ damage and many debilitating complications.
This therapy could be a huge game changer for patients struggling with sickle cell disease because of all of the complications that can occur with the condition. Sickled red blood cells can block the delivery of blood and oxygen to important organs and structures, which result in a wide array of symptoms and severe pain in the body. As an example, lack of blood flow to the bone can result in death to the bone, a condition called avascular necrosis.
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