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Sickle cell disease is a group of inherited disorders in which red blood cells — normally round — become hard, sticky and crescent shaped. The misshapen cells clog the flow of blood, which can ...
Samuel Charache (January 12, 1930 – January 29, 2019) was an American hematologist and professor at Johns Hopkins University. He led the research team that discovered the first effective treatment for sickle cell disease, a painful and sometimes fatal blood disorder that mainly affects people of African ancestry. [1]
Mendelian traits in humans. A 50/50 chance of inheritance. Sickle-cell disease is inherited in the autosomal recessive pattern. When both parents have sickle-cell trait (carrier), a child has a 25% chance of sickle-cell disease (red icon), 25% do not carry any sickle-cell alleles (blue icon), and 50% have the heterozygous (carrier) condition. [1]
A genetic basis for this disease was proposed in 1915 by Cook and Meyer. The disease was named sickle-cell anaemia in 1922 by Verne Mason after several additional cases were reported. All the known cases had been reported in blacks and he concluded that this disease was confined to those of black African descent.
Identifiers. DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1] [3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
Autosplenectomy caused by sickle cell anemia or hyposplenism in coeliac disease; In patients with obstructive liver disease, lecithin cholesterol acetyltransferase activity is depressed, which increases the cholesterol-to-phospholipid ratio and produces an absolute increase in the surface area of the red cell membrane.