Sickle Cell Anemia or also known as Sickle Cell Disease, is a severe hereditary form of anemia in which a mutated form of hemoglobin distorts the red blood cells into a crescent shape at low oxygen levels. When these red blood cells are affected in this way, the irregular shaped cells can get stuck in small blood vessels. If this happens, it can slow or block blood flow and oxygen to parts of the body. Symptoms of this disease easily affect people’s way of life, comfort, and overall characteristics of life. Fore example, victims of this disease have symptoms such as episodes of pain, painful swelling of hands and feet, frequent infections, delayed growth, and vision problems. This devastating disease affects 275,000 people globally, most commonly
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Usually, a bone marrow transplant is reserved for people younger than age sixteen because the risks increase for people older than sixteen. In addition, finding a donor is difficult, and the procedure has serious risks associated with it. Some tragic side-effects of this include infertility and for very few, death. The question is whether or not the risk of this procedure is worth it for some patients. To try and avoid such a gruesome path, researchers are looking at gene-related treatments. These therapies are being developed to try to correct the genetic abnormalities in patient’s own genes without swapping them out. This is usually done with the help of the gene-editing tool CRISPR, which they hope to be part of the future of curing sickle cell anemia. They have been able to alter genes in blood stem cells from patients with the disease and transplant them into mice and are trying to be approved to start new clinical trials to further test these methods. Another experimental alternative is a treatment of nitric oxide. People with the disease have low levels of nitric oxide in their blood. Nitric oxide is a gas that helps keep blood vessels open and reduces the stickiness of red blood cells. This might prevent the abnormal cells from clumping together, however, this has shown little benefit so far. Other types of potential treatments are not open to the public yet since they are undergoing FDA (U.S. Food and Drug Administration) review and it is confidential information. However, there are two drugs approved by the FDA to treat the disease: hydroxyurea and Endari, both of which work to reduce the frequency of “crises”, the severe attacks of pain and other complications as a result of this
Usually, a bone marrow transplant is reserved for people younger than age sixteen because the risks increase for people older than sixteen. In addition, finding a donor is difficult, and the procedure has serious risks associated with it. Some tragic side-effects of this include infertility and for very few, death. The question is whether or not the risk of this procedure is worth it for some patients. To try and avoid such a gruesome path, researchers are looking at gene-related treatments. These therapies are being developed to try to correct the genetic abnormalities in patient’s own genes without swapping them out. This is usually done with the help of the gene-editing tool CRISPR, which they hope to be part of the future of curing sickle cell anemia. They have been able to alter genes in blood stem cells from patients with the disease and transplant them into mice and are trying to be approved to start new clinical trials to further test these methods. Another experimental alternative is a treatment of nitric oxide. People with the disease have low levels of nitric oxide in their blood. Nitric oxide is a gas that helps keep blood vessels open and reduces the stickiness of red blood cells. This might prevent the abnormal cells from clumping together, however, this has shown little benefit so far. Other types of potential treatments are not open to the public yet since they are undergoing FDA (U.S. Food and Drug Administration) review and it is confidential information. However, there are two drugs approved by the FDA to treat the disease: hydroxyurea and Endari, both of which work to reduce the frequency of “crises”, the severe attacks of pain and other complications as a result of this