Eight weeks after CS-101 treatment, the patient's fetal hemoglobin (HbF) level has increased to ~95 g/L, accounting for ~81% of total hemoglobin. The percentage of HbF-expressing cells (F-cells ...

Data demonstrate therapeutically relevant editing levels using a clinically validated strategy, supporting its development as a novel, in vivo approach to treating sickle cell disease and beta ...

Fetal hemoglobin is naturally shut down after birth but can be reactivated by disrupting the BCL11A gene expression during erythropoiesis. “In β-thalassemia patients with hereditary persistence ...

The mutations are found in patients with a benign condition called ‘hereditary persistence of foetal haemoglobin’, or HPFH, in which the γ-globin that is normally switched off after birth ...

Hydroxyurea probably works by making it easier to make fetal hemoglobin again, Steinberg said. Many new therapies for sickle-cell disease focus on trying to increase fetal hemoglobin in the body.