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NPM1 small interfering RNA (siRNA) is a targeted molecular therapeutic designed to silence the expression of the Nucleophosmin (NPM1) gene through the RNA interference (RNAi) pathway. NPM1 is a multifunctional nucleolar protein that plays critical roles in ribosome biogenesis, centrosome duplication, and genomic stability; however, its overexpression or mutation (particularly the NPM1c+ mutation) is a hallmark of various malignancies, including acute myeloid leukemia (AML) and several solid tumors. By introducing synthetic double-stranded RNA sequences complementary to the NPM1 mRNA, this agent facilitates the assembly of the RNA-induced silencing complex (RISC), which subsequently cleaves the target mRNA and prevents protein translation. Preclinical studies have demonstrated that silencing NPM1 can inhibit cell proliferation, induce apoptosis, and enhance the sensitivity of cancer cells to conventional chemotherapeutic agents, making it a significant area of investigation in oncology.
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