Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

NFIX could be a target for sickle cell therapy

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.

Citation:

Shearstone JR et al. ASH 2019, Abstract 821.