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Diseases such as sickle cell disease that are caused by autosomal recessive disorders for which a person's normal phenotype or cell function may be restored in cells that have the disease by a normal copy of the gene that is mutated, may be a good candidate for gene therapy treatment.
What is sickle cell disease? Sickle cell disease, often called sickle cell anemia, which is its most serious form, is caused by a genetic disorder that alters the ability of red blood cells to ...
In sickle cell anemia, treatment is initially with intravenous fluids, pain medication, and oxygen therapy. [19] [3] The typical treatment of priapism may be carried out as well. [3] Blood transfusions are not usually recommended as part of the initial treatment, but if other treatments are not effective, exchange transfusion may be done. [19] [3]
Indications for red blood cell transfusion. Transfusion therapy for sickle-cell disease entails the use of red blood cell transfusions in the management of acute cases of sickle cell disease and as a prophylaxis to prevent complications by decreasing the number of red blood cells (RBC) that can sickle by adding normal red blood cells.
In a region that has the world’s highest rates of sickle cell, Lea Kilenga and her nonprofit are helping fellow Kenyans get crucial treatment and battling the stigma surrounding the disease.
Clark is involved with sickle cell disease awareness, research, treatment and programming in Pittsburgh. In 2012, he announced the formation of Ryan Clark's Cure League to raise awareness about sickle cell trait and eventually find a cure.
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