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Cardiovascular diseases (CVDs) are the leading causes of death globally and urgently require new novel therapeutic strategies. Gene therapy is the application of gene modulation technology to treat abnormal gene expression under disease conditions. Viral- and nonviral-based gene delivery systems are the foundation of gene modulation in target cells. Moreover, plasmid- or oligo-based gene modulation tools as well as new advancements in gene editing using CRISPR/Cas technology are currently being tested in a variety of clinical trials. Here, we summarized state-of-the-art gene therapy technologies as well as recent clinical trials and discuss the applications and lessons of gene therapy in CVDs., (© 2023. The Author(s), under exclusive license to Springer Nature Singapore Pte Ltd.)

Cardiovascular diseases are the leading cause of death globally and are associated with increasing financial expenditure. With the availability of next-generation sequencing technologies since the early 2000s, non-coding RNAs such as microRNAs, long non-coding RNAs and circular RNAs have been assessed as potential therapeutic targets for numerous diseases, including cardiovascular diseases. In this Review, we summarize current approaches employed to screen for novel coding and non-coding RNA candidates with diagnostic and therapeutic potential in cardiovascular disease, including next-generation sequencing, functional high-throughput RNA screening and single-cell sequencing technologies. Furthermore, we highlight viral-based delivery tools that have been widely used to evaluate the therapeutic utility of both coding and non-coding RNAs in the context of cardiovascular disease. Finally, we discuss the potential of using oligonucleotide-based molecular products such as modified RNA, small interfering RNA and RNA mimics/inhibitors for the treatment of cardiovascular diseases. Given that many non-coding RNAs have not yet been functionally annotated, the number of potential RNA diagnostic and therapeutic targets for cardiovascular diseases will continue to expand for years to come.