REGULATION OF TARGET GENE EXPRESSION AS A BREAKTHROUGH DIRECTION IN TREATMENT OF CARDIOVASCULAR DISEASES: FOCUS ON RNA THERAPY

Recent advances in the field of obtaining, purification and cellular delivery of RNA into the patient’s body have allowed to develop RNA-based therapeutic tools for treatment of a wide range of diseases, including cardiovascular ones. RNA therapy is a new, rapidly developing area of medicine that uses various RNA molecules as therapeutic agent. These medications are cost-effective, relatively easy to manufacture and can treat previously untreatable pathological processes. Currently, all RNA medications are divided into five groups and include antisense oligonucleotides (ASO), small interfering RNAs (siRNAs), microRNAs (miRNAs), RNA aptamers and mRNAs. RNA therapeutic drugs are designed to regulate the activity of genes and, depending on the chosen strategy, can replace, supplement, correct, suppress or eliminate the expression of the target gene. This mini review considers the challenges and advantages associated with the use of RNA-based medications, various approaches to their delivery to the patient’s cells, as well as the mechanisms of action of selected RNA medications. In addition, the review provides information on effectiveness of selected RNA-based drugs that are currently undergoing clinical trials or have already received regulatory approval.

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Автор(ы): K. A. Aitbaev, I. T. Murkamilov, V. V. Fomin, Z. F.Yusupova, T. F. Yusupova, F. A. Yusupov