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