The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …

Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

Systematic evaluation of the impact of genetic variants is critical for the study and treatment
of human physiology and disease. While specific mutations can be introduced by genome …

Reactivating silenced γ-globin expression through the disruption of repressive regulatory
domains offers a therapeutic strategy for treating β-hemoglobinopathies. Here, we used …

After many years of painstaking research, the potential of gene therapy to cure severe β-
thalassemia is now becoming evident through recent clinical trials. Therapeutic protocols …

Beta-hemoglobinopathies are the most common genetic disorders worldwide, caused by a
wide spectrum of mutations in the β-globin locus, and associated with morbidity and early …

Sickle cell disease (SCD) is one of the most common hemoglobinopathies. Due to its high
prevalence, with about 20 million affected individuals worldwide, the development of novel …

Sickle cell disease (SCD) is a hereditary blood disorder characterized by the presence of
abnormal hemoglobin molecules and thus distortion (sickling) of the red blood cells. SCD …

Ex vivo gene editing in hematopoietic stem and progenitor cells (HSPCs) represents a
promising curative treatment strategy for monogenic blood disorders. Gene editing using the …