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miR-124a precursor

Development stage
Preclinical
Lead developer
Kolling Institute of Medical Research
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral
01

Overview

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.

Other names
microRNA-124a precursormicroRNA124a precursormicroRNA 124a precursorpre-miR-124apre-miR124apre-miR 124ahsa-mir-124a precursorhsa-mir124a precursorhsa-mir 124a precursor
02

Targets

CDK6 (Cyclin-dependent kinase 6)PTBP1 (Polypyrimidine tract-binding protein 1)IQ motif containing GTPase activating protein 1 mRNA 3′-untranslated region miR-124a binding sitePHF19 (PHD finger protein 19)

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