mRNA-based Therapy for Neurodegenerative Diseases: Advantages and Limitations
https://doi.org/10.52667/2712-9179-2026-6-2-2-9
Abstract
mRNA-based therapy represents one of the most promising directions in modern biomedicine and is actively being investigated for the treatment of neurodegenerative diseases. In contrast to traditional gene therapy approaches based on DNA vectors, mRNA does not integrate into the host genome and does not require entry into the nucleus, thereby reducing the risk of mutagenesis and improving the safety profile of the method. This review provides an overview of the advantages and limitations of mRNA-based therapeutic approaches for neurodegenerative diseases. Key features of mRNA platforms are discussed, including the possibility of programmable expression of therapeutic proteins, the high flexibility of nucleotide sequence modification, and the relative simplicity of scalable production. Particular attention is given to mRNA delivery systems, especially lipid nanoparticles, which protect mRNA molecules from degradation and enhance the efficiency of intracellular delivery. The major limitations of this technology are also addressed, including the difficulty of crossing the blood-brain barrier, the low stability of mRNA, its potential immunogenicity, and the requirement for repeated administrations in the treatment of chronic diseases. In addition, current experimental strategies for the application of mRNA therapy are described, including protein replacement approaches, delivery of neuroprotective factors, and genome editing technologies. Overall, mRNA therapy holds considerable potential for the treatment of neurodegenerative disorders, however, its broad clinical implementation will require further improvements in delivery systems and enhanced stability of mRNA molecules.
Keywords
mRNA,
neurodegenerative diseases,
delivery systems,
lipid nanoparticles,
intracellular delivery,
neuroprotective factors
Supporting agencies: This work/publication was funded by a grant from the Academy of Sciences of the Republic of Tatarstan prvided to higher education institutions, scientific and other organizations to support human resource development plans in terms of encouraging their research and academic staff to defend doctoral dissertations and conduct research activities (Agreement No. 12/2025-PD-KFU dated December 22, 2025).
About the Authors
Aisylu I. Ayupova
Institute of Fundamental Medicine and Biology, Kazan Federal University
Russian Federation
Russian Federation
Kazan, 420008
Alexandra Sharshakova
Institute of Fundamental Medicine and Biology, Kazan Federal University
Russian Federation
Russian Federation
Kazan, 420008
Albert A. Rizvanov
Institute of Fundamental Medicine and Biology, Kazan Federal University;
Division of Medical and Biological Sciences, Tatarstan Academy of Sciences
Russian Federation
Russian Federation
Kazan, 420008
Kazan, 420111
Valeriya V. Solovyeva
Institute of Fundamental Medicine and Biology, Kazan Federal University
Russian Federation
Russian Federation
Kazan, 420008
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Review
For citations:
Ayupova A.I., Sharshakova A., Rizvanov A.A., Solovyeva V.V. mRNA-based Therapy for Neurodegenerative Diseases: Advantages and Limitations. Personalized Psychiatry and Neurology. 2026;6(2):2-9. https://doi.org/10.52667/2712-9179-2026-6-2-2-9
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