RNA CART123

RNA CART123 is an experimental, autologous CAR-T cell therapy developed by the University of Pennsylvania for the treatment of relapsed or refractory acute myeloid leukemia (AML). Unlike conventional CAR-T therapies that utilize viral vectors for permanent genetic modification, RNA CART123 employs RNA electroporation to transiently express a chimeric antigen receptor (CAR) targeting CD123 (Interleukin-3 receptor alpha). The CAR construct typically incorporates a CD123-specific single-chain variable fragment (scFv) linked to 4-1BB (CD137) costimulatory and CD3-zeta signaling domains. This transient expression approach is designed as a safety strategy to mitigate potential "on-target, off-tumor" toxicities, specifically the depletion of normal hematopoietic stem and progenitor cells that also express CD123. By using mRNA transfection rather than DNA integration, the therapeutic activity of the cells is limited to a few days, providing a "safety switch" through natural degradation of the CAR-encoding RNA.

Other names

CD123 Redirected Autologous T CellsCD-123 Redirected Autologous T CellsCD 123 Redirected Autologous T CellsRNA-electroporated anti-CD123 CAR T cellsmRNA-transfected anti-CD123 CAR-T cells