Article
Causal linkage of presence of mutant NPM1 to efficacy of novel therapeutic agents against AML cells with mutant NPM1.
Leukemia - 1 Jun 2023
Mill Christopher P, Fiskus Warren, Das Kaberi, Davis John A, Birdwell Christine E, Kadia Tapan M, DiNardo Courtney D, Daver Naval, Takahashi Koichi, Sasaki Koji, McGeehan Gerard M, Ruan Xinjia, Su Xiaoping, Loghavi Sanam, Kantarjian Hagop, Bhalla Kapil N
Abstract excerpt
In AML with NPM1 mutation causing cytoplasmic dislocation of NPM1, treatments with Menin inhibitor (MI) and standard AML chemotherapy yield complete remissions. However, the causal and mechanistic linkage of mtNPM1 to the efficacy of these agents has not been definitively established. Utilizing CRISPR-Cas9 editing to knockout (KO) or knock-in a copy of mtNPM1 in AML cells, present studies demonstrate that KO of...
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