MicroRNA-based integrated diagnosis and therapy for GBM: current status and advances

Glioblastoma (GBM) is the most common and aggressive type of central nervous system cancer, characterized by high rates of recurrence and mortality. As the highest-grade glioma, patient prognosis remains poor despite multimodal interventions including surgery, chemotherapy, and postoperative radiotherapy. Therefore, developing novel therapeutic strategies and precise diagnostic tools has become an urgent need in oncology research. In recent years, exosomes have emerged as important candidates for targeted tumor therapy due to their natural, endogenous nanocarrier properties, such as low immunogenicity, good biocompatibility, and the ability to cross biological barriers. In particular, exosome-based delivery systems loading functional microRNAs (miRNAs) offer a promising new strategy for intervening in malignant tumor progression. Studies have shown that exosome-delivered tumor-suppressive miRNAs can effectively inhibit tumor cell proliferation, promote apoptosis, impede migration and invasion, and reverse chemoresistance. These functions have been validated through in vitro cellular models and in vivo animal experiments across various tumors, confirming the efficacy of engineered exosome-miRNA delivery systems in suppressing tumor growth, delaying metastasis, and sensitizing tumors to treatment. Furthermore, in the field of biomarkers, aberrant expression of various miRNAs is closely associated with GBM proliferation, invasion, metastasis, and therapy resistance. Specifically, downregulated tumor-suppressive miRNAs and upregulated oncogenic miRNAs may serve as potential biomarkers for monitoring disease progression, assessing prognosis, and predicting therapeutic response. In summary, the miRNA system offers dual potential as both a targeted therapeutic approach and a precise biomarker, providing new directions for the diagnosis and treatment of GBM. However, challenges such as optimizing delivery efficiency and enhancing targeting specificity remain. Moving forward, interdisciplinary efforts will be essential to overcome these technical barriers and advance its translation from basic research to clinical application.