CD33KO-HSPC + CART33

CD33KO-HSPC + CART33 is a novel combination therapy approach for acute myeloid leukemia (AML) that consists of two components: 1) CD33 knockout hematopoietic stem and progenitor cells (CD33KO-HSPC) created using CRISPR/Cas9 gene editing, which are transplanted into patients to generate a CD33-negative myeloid system, and 2) chimeric antigen receptor T cells targeting CD33 (CART33), which are administered after successful engraftment to specifically eliminate CD33-positive leukemia cells while sparing the newly established CD33-negative normal myeloid cells. This approach allows for potent antigen-directed therapy without causing prolonged myeloid aplasia.