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Sickle cell-beta thalassemia is an inherited blood disorder. The disease may range in severity from being relatively benign and like sickle cell trait to being similar to sickle cell disease . [ 1 ] [ 2 ]
Gene therapy for hemoglobinopathies was first trialled in 2014 on a single patient with sickle cell disease (a fault in the beta globin gene), [93] and followed by clinical trials in which a number of patients with either sickle cell or beta thalassemia were successfully treated. [94]
The person has very few if any symptoms of sickle cell disease but carries the gene and can pass it on to their children. [56] Autosomal recessive inheritance means acquiring two changed genes from each parent. If both parents are carriers for the autosomal recessive gene, there is a 25% chance of their child having and expressing the disorder.
There are two clinically significant combinations involving the sickle cell gene: Hemoglobin S/ beta thalassemia: (see above). [35] Hemoglobin S/ hemoglobin C (Hemoglobin SC disease) occurs when an individual inherits one gene for hemoglobin S (sickle cell) and one gene for hemoglobin C, The symptoms are very similar to sickle cell disease. [37]
Sickle cell disease is a blood disorder wherein there is a single amino acid substitution in the hemoglobin protein of the red blood cells, which causes these cells to assume a sickle shape, especially when under low oxygen tension.
For people living with the disease, a sickle cell crisis can happen at any time. When it does, their rigid, sickle-shaped red blood cells become stuck in their blood vessels, blocking flow and ...
Sickle cell-Hb Lepore Boston syndrome is a type of sickle cell disease (HbS) that differs from homozygous sickle cell disease where both parents carry sickle hemoglobin. In this variant one parent has the sickle cell hemoglobin the second parent has Hb Lepore Boston, the only one of the three variants described in association with HbS. [7]
Splenic sequestration crisis is a life-threatening illness common in pediatric patients with homozygous sickle cell disease and beta thalassemia.Up to 30% of these children may develop splenic sequestration crisis with a mortality rate of up to 15%.
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