Retinoic acid and arsenic trioxide trigger degradation of mutated NPM1, resulting in apoptosis of AML cells

Key Points RA/arsenic induces proteasomal degradation of mutant NPM1, yielding AML growth arrest and apoptosis. RA/arsenic treatment restored nucleolar localization of NPM1 and significantly reduced bone marrow blasts in NPM1 mutant AML patients.