Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …

Gene editing to disrupt the GATA1-binding site at the+ 58 BCL11A erythroid enhancer could
induce γ-globin expression, which is a promising therapeutic strategy to alleviate β …

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

Background Sickle cell disease is characterized by hemolytic anemia, pain, and progressive
organ damage. A high level of erythrocyte fetal hemoglobin (HbF) comprising α-and γ …

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) …

For over 100 years, clinicians and scientists have been unravelling the consequences of the
A to T substitution in the β-globin gene that produces haemoglobin S, which leads to the …

To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we
investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or …

In the 100 years since sickle cell anemia (SCA) was first described in the medical literature,
studies of its molecular and pathophysiological basis have been at the vanguard of scientific …

Abstract β-Thalassemia and sickle cell disease (SCD) are the most prevalent monogenic
diseases. These disorders are caused by quantitative or qualitative defects in the production …