The genetic disorder I was told to research was the Sickle Cell Disease. I will explain what mutation causes this disease, the characteristics of it, and what has developed in the area of gene therapy because of it. The Sickle Cell Disease is an inherited disease. The gene for hemogoblin-S (which causes the disease) is the most common inherited blood condition in America; although most people only inherit one copy of the gene for HbS, while the other gene, hemogoblin-A, is normal, and can override HbS, blocking the disease. These people have the HbS trait, but not the disease, therefore leading a normal life. For an offspring to acquire the disease, both parents must have the HbS gene, yet the child only has a 25% chance of having Sickle Cells. You cannot catch the disease, you are born with it and it is prese
many research going on in the area of gene therapy. Labs around the world are trying to fix the basic genetic defect, by placing the correct amino acid in the hemogoblin before or shortly after birth. This method would result in the cure of the root of the problem. Currently researchers are finding a safe way to perform this method. To try to ease the pain caused by Sickle Cell Disease, a substance that can prevent red blood cells from sickling without causing harm to other parts of the body, hydroxyurea was found to reduce the frequency of severe pain, acute chest syndrome and the need for blood transfusions in adult patients with sickle cell disease. Droxia, the prescription form of hydroxyurea, was approved by the FDA in 1998 and is now available for adult patients with sickle cell anemia. Studies will now b
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