Stem Cell Gene Therapy for Sickle Cell Disease

Published 2023-09-04

In new research published in the New England Journal of Medicine, scientists from multiple acacemic children's hospitals including the University of Chicago took part in the a clinical trial using the CRISPR gene editing tool to disrupt the HBG1 and HBG2 (γ-globin) gene promoters. This lead to an increase in fetal hemoglobin expression in Red Blood Cells. Clinical trial participants experienced a significant decrease in vaso-occlusive events.