Key clinical point: Preclinical research suggests nuclear factor I X is a fetal hemoglobin repressor, a finding that could have implications for the treatment of sickle cell disease.
Major finding: Knocking down nuclear factor I X in adult erythroblasts induced fetal hemoglobin expression in 86%-97% of cells, and the total amount of fetal hemoglobin in those cells was 39%-40%.
Study details: Preclinical research on sorted erythroblast cell populations.
Disclosures: All researchers involved in this work are employees of Syros Pharmaceuticals.
Shearstone JR et al. ASH 2019, Abstract 821.