Therapeutic potential of microRNAs in neurological disorders: mechanisms, biomarkers, and emerging therapeutic strategies

MicroRNAs (miRNAs) are small, non-coding RNA molecules that play a pivotal role in post-transcriptional gene regulation, influencing various biological processes such as cell division, proliferation, and apoptosis. Recent research has illuminated the significant involvement of miRNAs in neurological disorders, which encompass a wide range of conditions affecting both the central and peripheral nervous systems. These disorders, including neurodegenerative diseases like Alzheimer’s and Parkinson’s, as well as psychiatric conditions such as depression and schizophrenia, impose a substantial burden on global health. Dysregulated miRNAs contribute to disease pathogenesis by modulating neuronal differentiation and related signaling cascades. This review explores the biogenesis of miRNAs and their dysregulation in neurological disorders, highlighting specific miRNAs that serve as potential biomarkers and therapeutic targets. For instance, decreased levels of miR-125b-5p and miR-26b-5p in cerebrospinal fluid have been associated with Alzheimer’s disease progression. In Parkinson’s disease, distinct profiles of dysregulated miRNAs have been identified, including miR-7-5p and miR-153-3p, which target α-synuclein. Furthermore, studies have demonstrated the potential of miRNA-based therapies to modulate disease processes and improve clinical outcomes. This review critically evaluates current therapeutic strategies for miRNA delivery in neurological disorders, focusing on advanced platforms such as nanocarriers, exosomes, viral vectors, and ligand-mediated systems designed to overcome the blood-brain barrier. We also explore the future of miRNA research in the context of precision medicine, highlighting the importance of targeted delivery, safety optimization, and integration of patient-specific molecular profiles. A comprehensive understanding of miRNA-regulated networks will be essential for developing innovative diagnostics and personalized treatments for neurodegenerative and neuroinflammatory diseases.