Prime Editing Corrects GATA2 Deficiency in Patient HSPCs

Company Aarhus University Event Type Peer-Reviewed Preclinical Publication (Science Translational Medicine) Modality Prime editing; PASSIGE (prime-editing-assisted site-specific integrase); ex vivo HSPC editing Asset Prime-edited autologous CD34-positive HSPCs (preclinical) Target GATA2 (c.956_962del variant) Disease Area GATA2 deficiency (GATA2 haploinsufficiency); inherited marrow failure and immunodeficiency Summary A…