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People who are successfully treated with gene editing therapy no longer have symptoms of sickle cell disease. This treatment is FDA-approved for people 12 years old and older. Long-term effects of this new treatment are not yet known and will continue to be studied.
Sickle cell anemia is one of a group of inherited disorders known as sickle cell disease. It affects the shape of red blood cells, which carry oxygen to all parts of the body. Red blood cells are usually round and flexible, so they move easily through blood vessels.
Sickle cell anemia. Symptoms & causes; Diagnosis & treatment; Doctors & departments
Can sickle cell disease be cured? Stem cell transplant is the only way to cure sickle cell disease. Stem cells are found in the bone marrow. The bone marrow is located inside the body’s bones and houses the blood cell factory. It’s where you find the gene mutation that causes sickle cell disease.
The Food and Drug Administration is expected to decide by Dec. 8 on a new therapy to treat sickle cell disease using gene editing technology called CRISPR, which stands for clustered regularly interspaced short palindromic repeats. Approximately 250 million people worldwide carry the gene for sickle cell disease.
With sickle cell disease, some red blood cells are shaped like sickles or crescent moons, become rigid and sticky. These sickle-cell shaped cells can slow or block blood flow. The most common type of sickle cell disease is sickle cell anemia.
If you have sickle cell disease, you might receive additional treatments that are used to treat disease-related episodes. Nonischemic Priapism Nonischemic priapism often goes away with no treatment.
Prompt treatment for priapism is usually needed to prevent tissue damage that could result in the inability to get or maintain an erection (erectile dysfunction). Priapism most commonly affects males in their 30s and older, but can begin in childhood for males with sickle cell disease.
Stem cell transplant. Also called a bone marrow transplant, a stem cell transplant might be an option in some cases. For children with severe thalassemia, it can eliminate the need for lifelong blood transfusions and drugs to control iron overload.
The U.S. Food and Drug Administration (FDA) has approved gene therapy products for several conditions, including cancer, spinal muscular atrophy, hemophilia and sickle cell disease. But for most people, gene therapy is available only as part of a clinical trial.