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Hematopoietic Stem Cell Transplant in Inherited Genetic Diseases

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Hematopoietic stem cell transplant (HSCT) is a curative treatment for many diseases especially inherited genetic diseases such as thalassemia, inherited bone marrow failure syndromes, primary immunodeficiencies, Gaucher disease, Hunter syndrome, or other mucopolysaccharidoses. The two main elements of HSCT are the conditioning regimen and an available donor. The conditioning regimen included combined chemotherapy, monoclonal antibody, or radiation, while the available donor might be HLA-identical or HLA-mismatched.

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