Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …

Iron is essential in many physiological processes, including DNA metabolism, oxygen
transport, and cellular energy generation. Deregulated iron metabolism, which results in iron …

Background Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to
reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short …

Summary Background Erythropoiesis-stimulating agents (ESAs) are the standard-of-care
treatment for anaemia in most patients with lower-risk myelodysplastic syndromes but …

Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …

β-Thalassemias Defective synthesis of the β-globin chain causes recessively inherited
disorders characterized by inadequate hemoglobin production and chronic anemia …

Transforming growth factor-β (TGF-β) signaling regulates multiple physiological processes,
such as cell proliferation, differentiation, immune homeostasis, and wound healing. Besides …

Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are the most
prevalent monogenic disorders worldwide. Trial HGB-205 (NCT02151526) aimed at …

Beta-thalassemia and particularly its transfusion-dependent form (TDT) is a demanding
clinical condition, requiring life-long care and follow-up, ideally in specialized centers and by …

The transforming growth factor beta (TGF-β) is a crucial cytokine that get increasing concern
in recent years to treat human diseases. This signal controls multiple cellular responses …