Inherited haemoglobin disorders, including thalassaemia and sickle-cell disease, are the
most common monogenic diseases worldwide. Several clinical forms of α-thalassaemia and …

Abstract β-Thalassemia is caused by reduced (β+) or absent (β 0) synthesis of the β-globin
chains of hemoglobin. Three clinical and hematological conditions of increasing severity are …

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

Patients with β-thalassemia major (BTM) require regular blood transfusions, supported by
appropriate iron chelation therapy (ICT), throughout their life. β-thalassemia is a global …

Abstract β-thalassemia is a hereditary hematological disease caused by over 350 mutations
in the β-globin gene (HBB). Identifying the genetic variants affecting fetal hemoglobin (HbF) …

Sickle cell anaemia (SCA) stands as a hereditary blood disorder characterized by mutated
haemoglobin, causing red blood cells to adopt a sickle shape, leading to complications like …

Non-transfusion-dependent thalassemias include a variety of phenotypes that, unlike
patients with beta (β)-thalassemia major, do not require regular transfusion therapy for …

Background In patients with non-transfusion-dependent β-thalassaemia, haemoglobin
concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality …

Red blood cells (RBC s) are generated from haematopoietic stem and progenitor cells
(HSPC s) through the step‐wise process of differentiation known as erythropoiesis. In this …

Beta-thalassaemia causes defective haemoglobin synthesis leading to ineffective
erythropoiesis, chronic haemolytic anaemia, and subsequent clinical complications. Blood …