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.

Editas Medicine’s in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to ...

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 ...