Abstract
Since the discovery of RNA interference (RNAi), excitement has grown over its potential therapeutic uses. Targeting RNAi pathways provides a powerful tool to change biological processes post-transcriptionally in various health conditions such as cancer or autoimmune diseases. Optimum design of shRNA, siRNA, and miRNA enhances stability and specificity of RNAi-based approaches whereas it has to reduce or prevent undesirable immune responses or off-target effects. Recent advances in understanding pathogenesis of autoimmune diseases have allowed application of these tools in vitro as well as in vivo with some degree of success. Further research on the design and delivery of effectors of RNAi pathway and underlying molecular basis of RNAi would warrant practical use of RNAi-based therapeutics in human applications. This review will focus on the approaches used for current therapeutics and their applications in autoimmune diseases, including rheumatoid arthritis and Sjögren’s syndrome.
Highlights
RNA interference (RNAi) is a cellular mechanism occurring in most eukaryotic cells that is responsible for post-transcriptional gene regulation
The effectors of the RNAi pathway are small RNA (RNA) molecules that can be classified into at least three categories based on their origin and function: microRNAs, short interfering RNAs, and short hairpin RNAs
MiRNAs represent endogenously encoded small RNAs, about 22 nucleotides long, which function to post-transcriptionally repress gene expression by binding to target mRNAs in a sequence specific manner
Summary
RNA interference (RNAi) is a cellular mechanism occurring in most eukaryotic cells that is responsible for post-transcriptional gene regulation This process involves small single stranded RNA molecules binding to the 3' UTR of specific mRNA targets resulting in the degradation or translational. MiRNAs represent endogenously encoded small RNAs, about 22 nucleotides long, which function to post-transcriptionally repress gene expression by binding to target mRNAs in a sequence specific manner. Pre-miRNA is exported from the nucleus via exportin-5, and is cleaved by Dicer in the cytoplasm to generate a mature miRNA about 22 nucleotides long This mature miRNA is incorporated into a complex known as the RNA-induced silencing complex (RISC) where it binds to its target mRNA though complementary base pairing. These molecules have become invaluable research tools to study the functions of genes and an alternative to knock-out mice
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