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HLA-Mismatched Hematopoietic Stem Cell Tranplantation for Pediatric Solid Tumors
Author(s) -
Andrea Pession,
Riccardo Masetti,
Corinne Di Leo,
Monica Franzoni,
Arcangelo Prete
Publication year - 2011
Publication title -
pediatric reports
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.297
H-Index - 19
ISSN - 2036-7503
DOI - 10.4081/pr.2011.s2.e12
Subject(s) - medicine , neuroblastoma , solid tumor , hematopoietic stem cell transplantation , human leukocyte antigen , oncology , pediatric oncology , stem cell , chemotherapy , hematopoietic stem cell , cancer , pediatric cancer , sarcoma , transplantation , haematopoiesis , immunology , pathology , genetics , antigen , biology , cell culture
The cure for thalassemia involves correcting the genetic defect in a hematopoietic stem cell that results in reduced or absent β-globin synthesis and an excess of α-globin dimers. Intracellular precipitation and accumulation of α- dimers results in ineffective erythropoiesis and hemolytic anemia. Replacing the abnormal thalassemic marrow with allogeneic normal or heterozygous stem cells carrying the functional gene restores appropriate β-globin chain synthesis

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