Reawakening ζ-Globin for Severe α-Thalassemia

Company Oxford University Event Type Publication / Peer-Reviewed Study Modality Base Editing (Promoter De-Repression) Asset HBZ Promoter-Editing Approach (Preclinical) Target HBZ (ζ-Globin) Promoter Elements Disease Area Severe α-Thalassemia (HbH Disease / Hb Bart’s Hydrops Fetalis) Summary An online-first, peer-reviewed Nature Genetics study maps two cis-regulatory…