Abstract
RNA therapeutics are emerging as a revolutionary approach for treating a wide spectrum of diseases by directly targeting underlying genetic and molecular mechanisms. RNA molecules offer unique therapeutic opportunities over small-molecule and peptide/protein-based drugs. These versatile molecules can be specifically directed to target disease-causing transcripts, which are otherwise difficult to target with small molecules or peptides/proteins. A variety of RNA therapeutics have been approved for medical use by the FDA. These include aptamers, antisense oligonucleotides, interfering RNAs, mRNA therapeutics, and RNA-guided CRISPR-Cas genome editing. Thus, RNA might be the next-generation drug to treat a variety of diseases. Unlike mRNA therapeutics, which restore or replace protein expression, and antisense oligonucleotides (ASOs), which modulate RNA splicing or promote transcript degradation, RNAi therapeutics mediate sequence-specific gene silencing through the RNA-induced silencing complex (RISC). These complementary RNA-based platforms provide distinct therapeutic strategies for treating a wide range of diseases. In this review, we discuss interfering RNAs, which have garnered increased attention and significance in recent times, particularly focusing on their role in cancer treatment. This review also discusses the future outlook in terms of the development of novel delivery vehicles, the role of artificial intelligence in the selection of RNAi, and the scope of RNAi in treating different types of diseases.