Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent
and severe monogenic disorder, SCD has been long considered a logical candidate for …

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

Distal enhancers commonly contact target promoters via chromatin looping. In erythroid
cells, the locus control region (LCR) contacts β-type globin genes in a developmental stage …

Naturally occurring, large deletions in the β-globin locus result in hereditary persistence of
fetal hemoglobin, a condition that mitigates the clinical severity of sickle cell disease (SCD) …

Abstract β-Thalassemia pathology is due not only to loss of β-globin (HBB), but also to
erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin …

In the present report, we carried out clinical-scale editing in adult mobilized CD34+
hematopoietic stem and progenitor cells (HSPCs) using zinc-finger nuclease-mediated …

Recently, gene therapy clinical trials have been successfully applied to
hemoglobinopathies, such as sickle cell disease (SCD) and β-thalassemia. Among the great …

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 …

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 …

Abstract β-Thalassemia intermedia is a clinical condition of intermediate gravity between β-
thalassemia minor, the asymptomatic carrier, and β-thalassemia major, the transfusion …