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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.
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 ...
Patients in the U.S. suffering from sickle cell disease can now receive targeted gene therapies using hematopoietic stem cells. [6] Hematopoietic stem cells are stem cells which differentiate and give rise to red blood cells, white blood cells and platelets. [7] These therapies involve removing hematopoietic stem cells from the patient and ...
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
A critical factor for healthy stem cell production is the bone marrow microenvironment. Important components are stromal cells, the extracellular matrix, and local cytokine gradients. The hematopoietic and non-hematopoietic elements of the bone marrow closely interact with each other and sustain and maintain the balance of hematopoiesis.
In chemotherapy as a conditioning regimen in hematopoietic stem cell transplantation, a study of people conditioned with cyclophosphamide alone for severe aplastic anemia came to the result that ovarian recovery occurred in all women younger than 26 years at time of transplantation, but only in five of 16 women older than 26 years. [116]