Stem Cell Gene Therapy for Sickle Cell Disease
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.
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