Sickle Cell Disease (SCD) is a hereditary blood disorder characterized by the presence of abnormal hemoglobin, known as hemoglobin S. This abnormality leads to the distortion of red blood cells into a rigid, crescent or “sickle” shape, especially under low oxygen conditions. These misshapen cells can cause blockages in small blood vessels, resulting in pain episodes, known as sickle cell crises, and various complications such as organ damage, increased risk of infections, and anemia. SCD is inherited in an autosomal recessive manner, meaning that an individual must inherit two copies of the sickle cell gene, one from each parent, to have the disease. Individuals with one sickle cell gene and one normal gene have a condition known as sickle cell trait, which typically does not cause symptoms but can be passed on to offspring. Treatment for SCD may include managing symptoms, pain relief, blood transfusions, and in some cases, bone marrow transplants.
In a groundbreaking move, the United States Food and Drug Administration (FDA) has given the green light to a revolutionary gene-editing treatment for sickle cell disease. This marks a significant milestone in the field of medical science, as it represents the first approval of cell-based gene therapies for this debilitating blood disorder. The FDA has […]
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