T cell-depleted hla-haploidentical stem cell transplantation in thalassemia young patients
| Autor: | Pietro Sodani, Antonella Isgrò, Javid Gaziev, Katia Paciaroni, Marco Marziali, Maria Domenica Simone, Andrea Roveda, Gioa De Angelis, Cristiano Gallucci, Fabio Torelli, Giancarlo Isacchi, Francesco Zinno, Fabiola Landi, Gaspare Adorno, Alessandro Lanti, Manuela Testi, Marco Andreani, Guido Lucarelli |
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| Jazyk: | angličtina |
| Rok vydání: | 2011 |
| Předmět: | |
| Zdroj: | Pediatric Reports, Pp e12-e12 (2011) |
| Druh dokumentu: | article |
| ISSN: | 2036-749X 2036-7503 |
| DOI: | 10.4081/pr.2011.s2.e12 |
| Popis: | 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. |
| Databáze: | Directory of Open Access Journals |
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