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miR-217 nanoparticle is an experimental RNA-based therapeutic being developed for the treatment of glioblastoma multiforme (GBM). It utilizes a nanoparticle delivery system to restore levels of microRNA-217 (miR-217), a tumor-suppressive miRNA that is frequently downregulated in GBM. miR-217 acts by directly targeting and inhibiting the expression of EZH2 (Enhancer of zeste homolog 2), the catalytic subunit of the Polycomb Repressive Complex 2 (PRC2). In GBM, EZH2-mediated H3K27 trimethylation maintains a cancer stem cell phenotype and represses tumor suppressor genes. By inhibiting EZH2, miR-217 nanoparticles re-activate these tumor suppressors, inhibit tumor growth, and enhance the efficacy of ionizing radiation.
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