MicroRNAs are small non-coding RNA molecules of 20–24 nucleotides in length. They play a key role in regulation of gene expression by influencing stability and translation of mRNA in a wide range of biological processes. Since their discovery, a large number of microRNAs have been described, and significant progress has been made in elucidating their functions. Convincing evidence now exists that microRNAs are powerful genetic regulators. The discovery of a link between microRNAs and numerous human diseases, particularly various types of cancer, stimulated a significant interest in exploring their therapeutic potential. They represent a new class of therapeutics capable of restoring impaired cellular functions, especially in various malignancies. Despite significant progress in preclinical research, microRNA-based therapy is still in the beginning stages; only a few microRNAs have been taken into further clinical trials. This is due to their off-target effects, primarily toxicity and immunogenicity, which significantly limit the use of microRNAs as therapeutic agents. One way to overcome off-target effects is to develop new systems for targeted delivery of microRNAs to the target tissue (disease site). Managing the off-target effects of microRNAs is a serious problem that requires solutions.
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Keywords: microRNA, microRNA therapy, microRNA delivery systems
Citation:
Kugaevskaya E.V., Timoshenko O.S., Gureeva T.A., Lisitsa A.V. (2026) MicroRNA: role in pathogenesis, problems of microRNA therapy, and future prospects. Biomeditsinskaya Khimiya, 72(3), 157-187.
Kugaevskaya E.V. et al. MicroRNA: role in pathogenesis, problems of microRNA therapy, and future prospects // Biomeditsinskaya Khimiya. - 2026. - V. 72. -N 3. - P. 157-187.
Kugaevskaya E.V. et al., "MicroRNA: role in pathogenesis, problems of microRNA therapy, and future prospects." Biomeditsinskaya Khimiya 72.3 (2026): 157-187.
Kugaevskaya, E. V., Timoshenko, O. S., Gureeva, T. A., Lisitsa, A. V. (2026). MicroRNA: role in pathogenesis, problems of microRNA therapy, and future prospects. Biomeditsinskaya Khimiya, 72(3), 157-187.
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