The β-thalassemias are a group of hereditary hematological diseases caused by over 300
mutations of the adult β-globin gene. Together with sickle cell anemia, thalassemia …

β-thalassaemia is a rare genetic condition caused by mutations in the β-globin gene that
result in severe iron-loading anaemia, maintained by a detrimental state of ineffective …

The hypoxia-inducible factor (HIF) pathway is crucial in mitigating the deleterious effects of
oxygen deprivation. HIF-α is an essential component of the oxygen-sensing mechanisms …

Gene editing by the CRISPR-Cas9 nuclease system technology can be considered among
the most promising strategies to correct hereditary mutations in a variety of monogenic …

Introduction: β-thalassemia is caused by autosomal mutations in the β-globin gene, which
induce the absence or low-level synthesis of β-globin in erythroid cells. It is widely accepted …

β-thalassemias are among the most common inherited hemoglobinopathies worldwide and
are the result of autosomal mutations in the gene encoding β-globin, causing an absence or …

A major goal of hemoglobinopathy research is to develop treatments that correct the
underlying molecular defects responsible for sickle cell disease and β-thalassemia. One …

Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …

Ineffective erythropoiesis is observed in many erythroid disorders including β‐thalassemia
and anemia of chronic disease in which increased production of erythroblasts that fail to …

The β-thalassemias are hereditary monogenic diseases characterized by a low or absent
production of adult hemoglobin and excess in the content of α-globin. This excess is …