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

URN to cite this document:
urn:nbn:de:bvb:355-epub-552369
DOI to cite this document:
10.5283/epub.55236
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.


Abstract

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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