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The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia
β-thalassemia is characterized by reduced or absence of β-globin production, resulting in anemia. Current therapies include blood transfusion combined with iron chelation. BM transplantation, although curative, is restricted by the matched donor limitation. Gene therapy, on the other hand, is promis...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
SAGE-Hindawi Access to Research
2011
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3236367/ https://www.ncbi.nlm.nih.gov/pubmed/22190966 http://dx.doi.org/10.4061/2011/987980 |