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Gene Therapy For Beta-Thalassemia: Updated Perspectives

1 Sept 2019

Abstract excerpt

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 represents a novel therapeutic promise, after many years of extensive preclinical research for the optimization of gene...

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PMID: 31576160
DOI: 10.2147/tacg.s178546

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