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Von Willebrand disease [8] Joint damage, potentially with severe pain and even destruction of the joint and development of arthritis: Hemophilia [7] Von Willebrand disease [8] Retinal bleeding: Acute leukemia [5] Transfusion transmitted infection, from blood transfusions that are given as treatment. Hemophilia [7] Adverse reactions to clotting ...
Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1]This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia.
Treatment is the same as for patients with sickle cell disease. Patients may receive hydroxyurea to induce the protective effects of increased fetal hemoglobin production. They may also benefit from blood transfusions especially during vaso-occlusive crises.
Iron-deficiency anemia is anemia caused by a lack of iron. [3] Anemia is defined as a decrease in the number of red blood cells or the amount of hemoglobin in the blood. [3] When onset is slow, symptoms are often vague such as feeling tired, weak, short of breath, or having decreased ability to exercise. [1]
There is no red cell effect with Ho-2 compared to Hemoglobin S, which changes the shape of the cell to become sickled. The Hopkins-2 variant of hemoglobin is not involved in forming sickle cells. There is a lack of phenotypic expression of Ho-2 in terms of sickle cell, so a person with sickle cell and hemoglobin Hopkins-2 would be asymptomatic.
Thalassemias are inherited blood disorders that result in abnormal hemoglobin. [7] Symptoms depend on the type of thalassemia and can vary from none to severe. [1] Often there is mild to severe anemia (low red blood cells or hemoglobin) as thalassemia can affect the production of red blood cells and also affect how long the red blood cells live. [1]
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