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) …

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

Featuring hundreds of full-color photomicrographs, Rodak's Hematology: Clinical Principles
and Applications, 5th Edition prepares you for a job in the clinical lab by exploring the …

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 …

The complex pathophysiology in β-thalassemia can translate to multiple morbidities that
affect every organ system. Improved survival due to advances in management means that …

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 …