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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 ...
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]
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...
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 ...
The human disease sickle cell anemia is caused by a genetic mutation in the codon for the sixth amino acid of the blood protein beta-hemoglobin.The normal DNA sequence G-A-G codes for the amino acid glutamate, while the mutation changes the middle adenine to a thymine, leading to the sequence G-T-G (G-U-G in the mRNA).
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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.
Sickle cell disorders, which are the most prevalent form of hemoglobinopathy. Sickle hemoglobin (HbS) is prone to polymerize when deoxygenated, precipitating within the red blood cell. This damages the RBC membrane resulting in its premature destruction and consequent anemia.
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