Prince C, Kumar D, Chambliss C, Okalava J, Malik S, Doering CB, Spencer HT, Archer D. Chandrakasan S (2025) Clinically relevant non-genotoxic conditioning with cd117 immunotoxin promotes robust donor chimerism and amelioration of sickle cell disease in a murine model. Transplantation and Cellular Therapy 31(2):S178. doi: 10.1016/j.jtct.2025.01.274
Objective: To investigate the efficacy of an Anti-CD117-Saporin conjugate as part of a non-genotoxic HCT strategy in a sickle cell disease (SCD) mouse model.
Summary: An Anti-CD117-SAP, combined with clinically relevant immunosuppression, achieved stable donor chimerism and corrected hematologic abnormalities in SCD mice. This conditioning regimen avoided transfusion requirements, graft-versus-host disease, and transplant-related mortality typical of TBI or busulfan-based approaches.
Usage: HbSS-Townes mice were conditioned with ATG and B cell depletion followed by Anti-CD117-SAP (IT-83, 0.75 µg/g) prior to HCT with HbAA-Townes donor marrow.
Related Products: Anti-CD117-SAP (Cat. #IT-83)