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Mucolipidosis II (I-cell disease) Fucosidosis; Aspartylglucosaminuria; Alpha-mannosidosis; Other Wolman disease (acid lipase deficiency) Immunodeficiencies. T-cell deficiencies Ataxia-telangiectasia; DiGeorge syndrome; Combined T- and B-cell deficiencies Severe combined immunodeficiency (SCID), all types; Well-defined syndromes Wiskott ...
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 ...
Treatments may include supportive care, drug therapy, and hematopoietic stem cell transplantation. [3] Supportive care may include blood transfusions, medications to increase the making of red blood cells, and antibiotics. [3] Drug therapy may include the medications lenalidomide, antithymocyte globulin, and azacitidine. [3]
Hematopoietic stem cells (HSCs) are the stem cells [1] that give rise to other blood cells.This process is called haematopoiesis. [2] In vertebrates, the first definitive HSCs arise from the ventral endothelial wall of the embryonic aorta within the (midgestational) aorta-gonad-mesonephros region, through a process known as endothelial-to-hematopoietic transition.
The first-line treatment of AML is usually chemotherapy, with the aim of inducing remission. [1] People may then go on to receive additional chemotherapy, radiation therapy, or a stem cell transplant. [1] [3] The specific genetic mutations present within the cancer cells may guide therapy, as well as determine how long that person is likely to ...
Stem-cell therapy uses stem cells to treat or prevent a disease or condition. [1] As of 2024, the only FDA-approved therapy using stem cells is hematopoietic stem cell transplantation. [2] [3] This usually takes the form of a bone marrow or peripheral blood stem cell transplantation, but the cells can also be derived from umbilical cord blood.
Younger individuals, if at high risk for dying from CLL, may consider allogeneic hematopoietic stem cell transplantation (HSCT). Myeloablative (bone marrow killing) forms of allogeneic stem cell transplantation, a high-risk treatment using blood cells from a healthy donor, may be curative, but treatment-related toxicity is significant. [80]
Clonal hematopoiesis of indeterminate potential, or CHIP, is a common aging-related phenomenon in which hematopoietic stem cells (HSCs) or other early blood cell progenitors contribute to the formation of a genetically distinct subpopulation of blood cells.