Beyond novel agents such as menin inhibitors or the nucleolar stress triggerer dactinomycin [23,24,25,26], which have shown anti-leukemic activity in preclinical models and are being investigated in clinical trials, cell therapy approaches targeting NPM1-mutated protein on AML cells could represent a useful complementary approach to the other available treatment options. The gene discussed is NPM1; the disease is acute myeloid leukemia.