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Defining curative endpoints for transfusion‐dependent β‐thalassemia in the era of gene therapy and gene editing

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Corbacioglu, Selim ; Frangoul, Haydar ; Locatelli, Franco ; Hobbs, William ; Walters, Mark
Date of publication of this fulltext: 21 Dec 2023 07:21

This publication is part of the DEAL contract with Wiley.


β-thalassemia is a monogenic disease that results in varying degrees of anemia. In the most severe form, known as transfusion-dependent β-thalassemia (TDT), the clinical hallmarks are ineffective erythropoiesis and a requirement of regular, life-long red blood cell transfusions, with the development of secondary clinical complications such as iron overload, end-organ damage, and a risk of early ...


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