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Sickle cell anemia is a classic example of the mixed benefit given by the staying power of pleiotropic genes, as the mutation to Hb-S provides the fitness benefit of malaria resistance to heterozygotes as sickle cell trait, while homozygotes have significantly lowered life expectancy—what is known as "heterozygote advantage". Since both of ...
Sickle cell disease (SCD), also simply called sickle cell, is a group of hemoglobin-related blood disorders that are typically inherited. [2] The most common type is known as sickle cell anemia. [2] Sickle cell anemia results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2]
The sickle cell trait provides a survival advantage against malaria fatality over people with normal hemoglobin in regions where malaria is endemic. The trait is known to cause significantly fewer deaths due to malaria, especially when Plasmodium falciparum is the causative organism.
The FDA is reviewing a drug that could cure sickle cell anemia. Right now bone marrow or stem cell transplants are the only FDA-approved cures. FDA advisers review sickle cell treatment that could ...
The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [10] [11] [12] 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. [13 ...
Red cell alloimmunisation is common in people with sickle cell disease who receive transfusions in Europe and North America. [4] This is because there are ethnic differences in the frequencies of blood group antigens. [4] Blood donors are usually Caucasian whereas the blood transfusion recipients usually have an African or Afro-Caribbean ancestry.
Clinical trials of gene therapy for sickle cell disease were started in 2014. [227] [228] In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the ...
Gene editing therapies aimed at increasing fetal hemoglobin production in beta-thalassemia as well as sickle cell anemia by inhibiting the BCL11A gene have been developed. [ 53 ] [ 54 ] Exagamglogene autotemcel , sold under the brand name Casgevy, is a gene therapy for the treatment of transfusion-dependent beta thalassemia developed by Vertex ...
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