Recent Advances in Molecular and Translational Medicine: Updates in Precision Medicine

Hematopoietic Stem Cell Transplant in Inherited Genetic Diseases

Author(s): Usanarat Anurathapan* and Suradej Hongeng

Pp: 225-234 (10)

DOI: 10.2174/9789815036756121010015

* (Excluding Mailing and Handling)

Abstract

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.


Keywords: Conditioning Regimen, Fanconi Anemia, Hematopoietic Stem Cell Transplantation, Human Leukocyte Antigen, Severe Combined Immunodeficiency Syndrome, Thalassemia.

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