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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 ...
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 ...
Malignant melanoma of the extremities, multiple myeloma, conditioning treatment before haemopoietic stem cell transplant. Myelosuppression, pulmonary fibrosis and pneumonitis (uncommon), skin necrosis (uncommon), anaphylaxis, hepatic sinusoidal obstruction syndrome and SIADH. Secondary malignancies. [16] Streptozotocin: IV, PO: Alkylates DNA.
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.
The gene therapy is made from the recipient's own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. [11] Prior to treatment, the recipient's own stem cells are collected, and then the recipient must undergo myeloablative conditioning (high-dose ...
Haematopoietic stem cell transplantation (HSCT) is the transplantation of multipotent haematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood. [ 10 ] [ 11 ] [ 12 ] It may be autologous (the patient's own stem cells are used), allogeneic (the stem cells come from a donor) or syngeneic (from an ...
It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in May 2018. [5] Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells. [6] It was approved for medical use by the U.S. Food and Drug Administration in September 2022 ...
Hematopoietic stem cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood. [ 16 ] [ 17 ] [ 13 ] It may be autologous (the patient's own stem cells are used), allogeneic (the stem cells come from a donor) or syngeneic (from an ...
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