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Gene Therapy For Beta-Thalassemia: Updated Perspectives

Allogeneic hematopoietic stem cell transplantation was until very recently, the only permanent curative option available for patients suffering from transfusion-dependent beta-thalassemia. Gene therapy, by autologous transplantation of genetically modified hematopoietic stem cells, currently represe...

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Dades bibliogràfiques
Publicat a:Appl Clin Genet
Autors principals: Karponi, Garyfalia, Zogas, Nikolaos
Format: Artigo
Idioma:Inglês
Publicat: Dove 2019
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6765258/
https://ncbi.nlm.nih.gov/pubmed/31576160
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TACG.S178546
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