Work

The Role of MicroRNAs in Glioblastoma: Gene Therapy and Diagnostic Potentials

Year: 2025
Type: article
Study design: Systematic Review
Abstract: Objective: MicroRNAs (miRNAs) are a class of small, non-coding RNA molecules, typically 18–25 nucleotides in length, that regulate gene expression post-transcriptionally by binding to complementary sequences in messenger RNAs (mRNAs), leading to mRNA degradation or translational repression. They are endogenously expressed and are transcribed from DNA but do not encode proteins. Instead, they play a crucial role in fine-tuning cellular processes such as development, differentiation, apoptosis, and metabolism. miRNAs are usually identified through next-generation sequencing (NGS), microarray analysis, or real-time quantitative PCR (RT-qPCR). Synthetic miRNAs and miRNA inhibitors (antagomiRs) can also be engineered in the laboratory for research and therapeutic purposes. These molecules are often delivered via viral vectors, lipid nanoparticles, or exosome-based systems in preclinical and clinical studies. Over the past decade, miRNAs have become established as important molecular tools in both research and clinical settings. They are routinely used as biomarkers for disease diagnosis, prognosis, and therapeutic monitoring, particularly in oncology, cardiovascular diseases, and neurological disorders. Due to their stability in body fluids such as blood, cerebrospinal fluid, and urine, circulating miRNAs are being actively explored for use in minimally invasive diagnostic approaches, including liquid biopsies. Material and Methods: A systematic review was conducted using established protocols including identification, screening, eligibility assessment, data extraction, and best practices in evaluation. Literature was retrieved from databases such as PubMed, Scopus, EBSCO, EMBASE, LILACS, ScienceDirect, and Web of Science for the period between January 2020 and March 2025. The most relevant and high-quality studies on miRNAs were included in the analysis. Results: A total of 42 high-quality studies were selected following a systematic review process based on PRISMA guidelines. The findings highlight the multifaceted roles of miRNAs in glioblastoma, including the regulation of gene expression, suppression of oncogenes, modulation of tumor suppressor pathways, and enhancement of chemosensitivity. Several miRNAs (e.g., miR-21, miR-128-3p, miR-522-3p) were identified as key modulators in glioblastoma biology, with documented effects on tumor progression, angiogenesis, and treatment resistance. Furthermore, miRNAs demonstrated promising utility as diagnostic biomarkers due to their stability in body fluids and ability to reflect tumor-specific molecular alterations. Their delivery through nanocarriers and exosomes was also explored, although challenges such as immunogenicity, off-target effects, and blood–brain barrier penetration remain to be fully addressed. Conclusion: miRNAs represent a powerful class of regulatory molecules with significant implications for the diagnosis, prognosis, and treatment of glioblastoma. Their ability to modulate gene expression at multiple levels, particularly through the suppression of oncogenic pathways and the enhancement of tumor suppressor mechanisms, underscores their therapeutic potential. In addition, their detectability in body fluids and involvement in various tumor-specific pathways make them attractive candidates for non-invasive diagnostic applications. While challenges such as delivery efficiency, specificity, and safety remain, ongoing advancements in nanotechnology and exosome-based systems offer promising avenues for clinical translation. Future research should focus on validating specific miRNA targets, optimizing delivery strategies, and integrating miRNA profiling into personalized treatment approaches for glioblastoma.
Institution: Hamdard University
Language: English
Subfield: Cancer Research
Open Access status: diamond
OpenAlex ID: W4410059557
FWCI: 2.019
Cites:
Cited by: 6
Related to: 10