This review provides an overview of significant developments in RNA-based therapeutics. Therefore, RNA-based therapies can broaden the range of druggable targets and are regarded as the most attractive therapeutic target. RNA aptamers can also block protein activity, similar to small-molecule inhibitors and antibodies. Moreover, clustered regularly interspaced short palindromic repeat (CRISPR)-based genome editing can directly modify target RNA sequences to treat specific disorders. This process would not cause irreversible genome changes and induce genetic risks like DNA-based therapeutics. In addition, in vitro transcribed (IVT) mRNA can be applied for protein replacement treatment or immunization after entering the cytoplasm. Besides, around 85% of proteins lack specific clefts and pockets for small molecules binding. By contrast, only 0.05% of the human genome has been drugged by the currently approved protein-targeted therapeutics (small-molecule chemicals and antibodies) since most DNA sequences of the human genome are transcribed into noncoding transcripts. Therefore, RNA can theoretically target any interest gene by selecting the correct nucleotide sequence on the target RNA. RNA molecules such as ASOs, small interfering RNA (siRNAs), and microRNAs (miRNAs) can directly target mRNAs and noncoding RNAs (ncRNAs) through Watson–Crick base-pairing. RNAs function in three essential biological macromolecules: DNAs, RNAs, and proteins. Compared to conventional protein-targeted and DNA-based medicines, RNA-based therapeutics are prospective due to their distinct physicochemical and physiological properties.
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