A novel compound CAR-T targeting both CD33 and CLL-1 was tested in a phase I study in which a 6 year-old female with Fanconi’s anemia-associated juvenile myelomonocytic leukemia carrying FLT3-ITD mutation that had been heavily treated with multiple therapies, including a FLT3 inhibitor [127]. The gene discussed is FLT3; the disease is Fanconi anemia.