Background Sickle cell disease is caused by a defect in the β-globin subunit of adult
hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed …

Hereditary anemia has various manifestations, such as sickle cell disease (SCD), Fanconi
anemia, glucose-6-phosphate dehydrogenase deficiency (G6PDD), and thalassemia. The …

Sickle cell disease (SCD) and b-thalassemia are the most common monogenic disorders in
the world. Their incidence is expected to increase over time, primarily in low-or middle …

Sickle cell disease (SCD) is potentially curable after allogeneic hematopoietic stem cell
transplantation (HSCT) or autologous HSCT after ex vivo genetic modification. Autologous …

Recent studies suggest that plerixafor mobilization and apheresis in patients with sickle cell
disease (SCD) is safe and can allow collection of sufficient CD34+ hematopoietic stem cell …

Sickle cell disease (SCD) is caused by a 20A> T mutation in the β-globin gene. Genome-
editing technologies have the potential to correct the SCD mutation in hematopoietic stem …

Sickle cell disease (SCD) is an exemplar of bidirectional translational research, starting with
a remarkable astute observation of the abnormally shaped red blood cells that motivated …

Sickle cell disease (SCD) is caused by a well-defined point mutation in the β-globin gene
and therefore is an optimal target for hematopoietic stem cell (HSC) gene-addition/editing …

Sickle cell disease (SCD) is a monogenic disorder characterized by recurrent episodes of
severe bone pain, multi-organ failure, and early mortality. Although medical progress over …

Thalassemia and sickle cell disease (SCD) are the most common monogenic diseases in
the world and represent a growing global health burden. Management is limited by a paucity …