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Sickle cell disease ( SCD ), also simply called sickle cell, is a group of hemoglobin-related blood disorders typically inherited. [2] The most common type is known as sickle cell anemia. [2] It results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2] This leads to a rigid, sickle -like shape under ...
Hematology. Sickle cell trait describes a condition in which a person has one abnormal allele of the hemoglobin beta gene (is heterozygous ), but does not display the severe symptoms of sickle cell disease that occur in a person who has two copies of that allele (is homozygous ). Those who are heterozygous for the sickle cell allele produce ...
Asplenia. Asplenia. Specialty. Medical genetics. Asplenia refers to the absence of normal spleen function and is associated with some serious infection risks. Hyposplenism is used to describe reduced ('hypo-') splenic functioning, but not as severely affected as with asplenism. Functional asplenia occurs when splenic tissue is present but does ...
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.
Angella Dorothea Ferguson was born in Washington, D.C. to George Alonzo Ferguson and his wife Mary Burton Ferguson. She is African-American and was one of eight children. [3] Though her father was a high school teacher, had his own architectural firm, and was a U.S. Army reservist, the family struggled financially, especially during the Great ...
Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. [1] [2] [3] The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved ...
Achieving success in sickle cell disease will illustrate that with effective global partnerships, inequities in biomedical science and health can be addressed. Fellowships and other awards. Makani received a training (2003) and intermediate fellowship (2011) from the Wellcome Trust for the sickle cell disease programme.
Stem-cell transplantation was pioneered using bone marrow-derived stem cells by a team at the Fred Hutchinson Cancer Research Center from the 1950s through the 1970s led by E. Donnall Thomas, whose work was later recognized with a Nobel Prize in Physiology or Medicine. Thomas' work showed that bone-marrow cells infused intravenously could ...