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mRNA therapy effective treatment of rare hereditary diseases
https://doi.org/10.30629/0023-2149-2024-102-5-6-410-414
Abstract
mRNA therapy, or mRNA-based drugs that have emerged thanks to vaccines against SARS-CoV-2, have successfully passed preclinical tests and are currently at various stages of clinical trials in the treatment of many diseases, including rare metabolic disorders. In the case of rare genetic metabolic diseases, the concept of mRNA therapy can be considered as an alternative to protein replacement therapy, where exogenous mRNA leads to the production of a fully active protein instead of a non-functional one, and also delivers it to the desired cellular compartment, such as mitochondria or the cell membrane. Preclinical studies on animal models of some rare genetic diseases have fully confrmed the validity of this concept. In this mini-review, we examine and discuss the mentioned preclinical studies on efficacy and safety in several animal models. For all the diseases considered, mRNA therapy restored functional protein to therapeutically significant levels in target organs, led to stable and reproducible results after each dose of mRNA, and was well tolerated, as confirmed by functional liver tests evaluated in animal models, including non-human primates. These data convincingly confirm the potential of clinical development of mRNA therapy for the treatment of various rare metabolic disorders.
About the Authors
K. A. Aitbaev
Scientific and Research Institute of Molecular Biology and Medicine
Kyrgyzstan
Kyrgyzstan
Kubanych A. Aitbaev — Doctor of Medical Sciences, Professor, Head of the Laboratory of Pathological Physiology and Immunology, member of the Board of the Association of Specialists for Chronic Kidney Disease in Kyrgyzstan
Bishkek
I. T. Murkamilov
I.K. Akhunbaev Kyrgyz State Medical Academy; Kyrgyz-Russian Slavic University named after the First President of the Russian Federation B.N. Yeltsin
Kyrgyzstan
Kyrgyzstan
Ilkhom T. Murkamilov — Doctor of Medical Sciences, Associate Professor at the Department of Internal Medicine; Associate Professor of Therapy No. 2 at the Medical Faculty, Chairman of the board of the Association of Specialists in Chronic Kidney Disease in Kyrgyzstan
Bishkek
V. V. Fomin
I.M. Sechenov First Moscow State Medical University of the Ministry of Health of Russia (Sechenov University)
Russian Federation
Russian Federation
Viktor V. Fomin — Doctor of Medical Sciences, Professor, Corresponding Member of the Russian Academy of Sciences, Head of the Department of Faculty Therapy №1 at N. V. Sklifosovsky Institute of Clinical Medicine, Vice-Rector for Innovation and Clinical Activity
Moscow
F. A. Yusupov
Osh State University
Kyrgyzstan
Kyrgyzstan
Furkat A. Yusupov — Doctor of Medical Sciences, Professor, Head of the Department of Neurology, Neurosurgery and Psychiatry at the medical faculty, member of the Board of the Association of Specialists for Chronic Kidney Disease in Kyrgyzstan and chief neurologist in the southern region of Kyrgyzstan
Osh
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Review
For citations:
Aitbaev K.A., Murkamilov I.T., Fomin V.V., Yusupov F.A. mRNA therapy effective treatment of rare hereditary diseases. Clinical Medicine (Russian Journal). 2024;102(5-6):410-414. (In Russ.) https://doi.org/10.30629/0023-2149-2024-102-5-6-410-414
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