This article reviews RNA therapies targeting cardiovascular diseases, highlighting efficacy and challenges.
RNA therapeutic agents represent a novel and promising class of drugs aimed at preventing and treating various diseases, including cardiovascular pathologies. These agents enable temporary regulation of target gene expression, opening new possibilities for therapy. Depending on the approach, RNA-based drugs can replace, supplement, correct, or suppress gene expression. The main classes of RNA therapy include antisense oligonucleotides (ASOs), microRNAs (miRNAs), small interfering RNAs (siRNAs), RNA aptamers, and messenger RNAs (mRNAs). This article discusses existing challenges, such as drug delivery to target organs, as well as the advantages of RNA therapy, including cost-effectiveness, simplicity of production, and the ability to target hard-to-treat diseases. Additionally, the mechanisms of action of various RNA-based drugs and their impact on gene expression are thoroughly reviewed. Particular attention is given to clinical trials and the efficacy of drugs that have already received regulatory approval.
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Айтбаев et al. (2025) studied this question.
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