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The FDA is reviewing a new drug for sickle cell disease that uses CRISPR to edit DNA. Black patients with the disease are excited and hopeful about the treatment.
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]
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]
The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [9] [10] [11] It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. [8 ...
Peripheral blood smear in patient with thrombotic thrombocytopenic purpura. Typical schistocytes are annotated. A schistocyte or schizocyte (from Greek schistos for "divided" and kytos for "hollow" or "cell") is a fragmented part of a red blood cell.
Sickle-cell disease was the genetic disorder to be linked to a mutation of a specific protein. Pauling introduced his fundamentally important concept of sickle cell anemia as a genetically transmitted molecular disease. [20] This vein (4) shows the interaction between the malaria sporozoites (6) with sickle cells (3) and regular cells (1).
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