Abstract

MicroRNAs (miRNAs) are small non-coding RNAs 18–24 nucleotides long and function as a post-transcriptional regulator of the expression of protein-coding target genes. It has been proven that normally microRNAs play an important role in various biological processes, including proliferation, differentiation, and apoptosis. Importantly, dysregulation of miRNAs is found to be involved in the pathogenesis of various human tumors, including brain tumors. Throughout the world, the problem of morbidity and mortality associated with brain tumors (e.g., glioblastomas multiforme (GBM)) has occupied a leading position for many years. Modern treatment strategies are based on surgery, chemotherapy, and radiation therapy. However, none of these treatments, alone or in combination, is considered effective. The data show that miRNAs can act as both a suppressor and an oncogene of tumor growth, regulating the processes of proliferation, tumor invasion, apoptosis, angiogenesis, immune response, metastasis, and drug resistance. While discussing recent studies targeting miRNAs to treat neuro-oncological conditions, we will discuss the advantages and possible limitations of miRNA-based gene therapy, the feasible methods for miRNA-based gene delivery, and the clinical therapeutic prospects of miRNA-based gene therapy for brain tumors.