By SPC News Staff
Will gene therapy someday be used to help patients with sickle cell disease (SCD)? That is the hope of researchers who are converting a pathogenic hemoglobin gene to a benign variant that enables healthy blood cell production in an animal model of SCD.
A team led by researchers at the Broad Institute of MIT and Harvard and St. Jude Children’s Research Hospital demonstrated a base editing approach that efficiently corrects the mutation underlying SCD in patient blood stem