Article
Small interfering RNA-mediated silencing of mutant NPM1 suppresses acute myeloid leukemia via reversing KAT7 and p300-mediated histone acetylation.
Leukemia - 1 Aug 2026
Hou Yongqiang, Xing Yunzhi, Chen Chunjie, Su Dongxue, Zhou Zhien, Ye Yi, Lai Zhangjian, Peng Zhixian, Cai Hongxi, Sun Min, Liu Yue, Yang Bingying, Huang Haitao, Li Zhe, Li Jinhuan, Li Junhong, Gao Huan, Li Jiaxin, Zhao Hao, Shi Yiran, Chen Qinghua, Long Jun, Hu Jiong, Zhou Dawang, Chen Lanfen
Abstract excerpt
NPM1 mutation (NPM1c)-driven acute myeloid leukemia (AML) is characterized by the sequestration of nuclear proteins and chromatin hijacking. Current treatment strategies targeting NPM1c AML are often indirect, which might lead to toxicity and resistance. In this study, we developed an allele-specific siRNA that selectively silences NPM1c while preserving the function of wild-type NPM1. This approach inhibited...
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