American Society of Hematology, Blood, 22(125), p. 3447-3454, 2015
DOI: 10.1182/blood-2014-11-612416
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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.