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Hematopoietic Stem Cell Transplant in Inherited Genetic Diseases

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

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.

Original languageEnglish
Title of host publicationRecent Advances in Molecular and Translational Medicine
Subtitle of host publicationUpdates in Precision Medicine
PublisherBentham Science Publishers
Pages225-234
Number of pages10
ISBN (Electronic)9789815036756
ISBN (Print)9789815036763
DOIs
Publication statusPublished - 1 Jan 2021

Keywords

  • Conditioning Regimen
  • Fanconi Anemia
  • Hematopoietic Stem Cell Transplantation
  • Human Leukocyte Antigen
  • Severe Combined Immunodeficiency Syndrome
  • Thalassemia

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