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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 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.
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 identical twin).
(The one-year survival rate has been estimated to be roughly 60%, although this figure includes deaths from the underlying disease, as well as from the transplant procedure.) [51] Major complications include veno-occlusive disease, mucositis, infections , graft-versus-host disease, and the development of new malignancies.
Cell therapy (also called cellular therapy, cell transplantation, or cytotherapy) is a therapy in which viable cells are injected, grafted or implanted into a patient in order to effectuate a medicinal effect, [1] for example, by transplanting T-cells capable of fighting cancer cells via cell-mediated immunity in the course of immunotherapy, or ...
Peripheral blood stem cell transplantation (PBSCT), also called "Peripheral stem cell support", [1] is a method of replacing blood-forming stem cells.Stem cells can be destroyed through cancer treatments such as chemotherapy or radiation, as well as any blood-related diseases, such as leukemia, lymphoma, neuroblastoma and multiple myeloma. [2]
Hematopoietic stem cell transplantation (HSCT) is the transplantation of blood stem cells derived from the bone marrow (that is, bone marrow transplantation) or blood. Stem cell transplantation is a medical procedure in the fields of hematology and oncology, most often performed for people with diseases of the blood, bone marrow, or certain ...
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.