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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 ...
Hematopoietic stem-cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood, in order to replicate inside a patient and produce additional normal blood cells.
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia which adds a healthy beta-globin gene to the HSCs. [97] It was approved for medical use in the United States in August 2022. [95] [98] The procedure involves collecting hematopoietic stem cells (HSCs
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 ...
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.
Irreversible stem cell damage in the bone marrow is the first life-threatening effect of intense radiation exposure and therefore one of the most important bodily elements to protect. Due to the regenerative property of hematopoietic stem cells , it is only necessary to protect enough bone marrow to repopulate the exposed areas of the body with ...
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 ...
The treatment is personalized for each person; hematopoietic stem cell (HSCs) are extracted from the person and purified so that only CD34-expressing cells remain.Those cells are cultured with cytokines and growth factors and then transduced with a gammaretrovirus containing the human adenosine deaminase gene and then reinfused into the person.