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Targeting a cell surface RNA-binding protein driving acute myeloid leukemia

How modifications to RNA molecules and the proteins they interact with on the cell surface contribute to cancer is largely unknown. Preclinical evidence indicates that cell surface nucleophosmin (NPM1, an RNA-binding protein) is a novel druggable biomarker in acute myeloid leukemia, with potential implications for improving detection and immunotherapy strategies for several cancer types.

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Fig. 1: Nanoscale distribution of cell surface NPM1 and its therapeutic effect on AML in vivo.

References

  1. Box, J. K. et al. Nucleophosmin: from structure and function to disease development. BMC Mol. Biol. 17, 19 (2016). A review article that summarizes the biology of NPM1 and its relevance in disease.

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  2. Perr, J. et al. RNA-binding proteins and glycoRNAs form domains on the cell surface for cell-penetrating peptide entry. Cell 188, 1878–1895.e25 (2025). This paper reports our characterization of cell surface RBPs and their role in regulating cell entry of cell-penetrating peptides.

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This is a summary of: George, B. M. et al. Treatment of acute myeloid leukemia models by targeting a cell surface RNA-binding protein. Nat. Biotechnol. https://doi.org/10.1038/s41587-025-02648-2 (2025).

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Targeting a cell surface RNA-binding protein driving acute myeloid leukemia. Nat Biotechnol (2025). https://doi.org/10.1038/s41587-025-02695-9

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  • DOI: https://doi.org/10.1038/s41587-025-02695-9

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