Development of CAR T Cells Targeting a Surface RNA Binding Protein for the Treatment of Acute Leukemias

Researchers generated CAR-T cells against one antigen – U5 snRNP200, an RNA helicase localized to the acute myeloid leukemia (AML) cell surface and absent from normal hematopoietic precursors. Anti-U5 snRNP200 CAR-T cells were effective in human and syngeneic models of AML as well as B cell acute lymphoblastic leukemia.