Selami Demirci

Oct 12, 2023 | nature.com | Naoya Uchida |Selami Demirci |Xiaolin Wu |Cynthia E. Dunbar |Robert E. Donahue

AbstractHematopoietic stem cell (HSC) gene therapy has curative potential; however, its use is limited by the morbidity and mortality associated with current chemotherapy-based conditioning. Targeted conditioning using antibody-drug conjugates (ADC) holds promise for reduced toxicity in HSC gene therapy. Here we test the ability of an antibody-drug conjugate targeting CD117 (CD117-ADC) to enable engraftment in a non-human primate lentiviral gene therapy model of hemoglobinopathies.