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miR-124a precursor is a synthetic microRNA molecule designed to mimic the function of endogenous miR-124a, a microRNA that is highly expressed in normal brain tissue but significantly downregulated in glioblastoma multiforme (GBM). It acts as a tumor suppressor by targeting the 3'-untranslated region (UTR) of specific oncogenic mRNAs, most notably IQGAP1, which is a key regulator of cell migration and invasion. By restoring miR-124a levels, the precursor inhibits the expression of IQGAP1 at both the mRNA and protein levels, thereby reducing the invasive and migratory capacity of GBM cells. Preclinical studies have demonstrated that delivery of this precursor can limit tumor spread, suggesting its potential as a novel therapeutic approach for invasive brain tumors, either as a monotherapy or in combination with conventional chemotherapy.
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