This has been enabled by noncoding RNAs (ncRNAs), which were once thought of as transcriptional noise but have turned out to be potent gene expression controllers and effective therapeutic targets. These non-protein-coding RNAs consist of microRNAs (miRNAs), small interfering RNAs (siRNAs), and long noncoding RNAs (lncRNAs). The abnormal functioning of ncRNAs has been closely associated with cancer, cardiovascular diseases, neurodegenerative disorders, and metabolic diseases. Therapy has been directed towards the inhibition of disease-causing non-coding RNAs (ncRNAs) or the restoration of protective ones. siRNA-based drugs are able to selectively silence disease-causing genes, whereas miRNA mimics and inhibitors can regulate the function of whole gene networks. The recent development of chemical modification and nanoparticle delivery systems has enhanced the stability, tissue targeting, and safety of ncRNA therapeutics. Several RNA-based drugs received regulatory approval, which confirms their clinical potential. As compared to traditional drugs, ncRNA therapeutics are highly specific and less off-target. Nevertheless, such challenges as immune activation, delivery efficiency, and long-term safety are still being actively studied. These platforms are still under continuous research and development to establish the noncoding RNAs as one of the pillars of the next generation, precision-based therapeutics.
MBH/PS