Key result
Antisense oligonucleotides and RNA interference represent promising therapeutic strategies for inflammatory bowel disease, though large clinical trials have yet to confirm the efficacy seen in preclinical models.
Why the study?
Crohn's disease and ulcerative colitis lack curative therapies, and advances in molecular biology have enabled the development of antisense oligonucleotides that selectively target molecules involved in IBD pathogenesis.
While antisense oligonucleotide and RNA interference technologies offer targeted approaches for IBD treatment, clinical translation remains challenging due to delivery and efficacy issues.
ASO and RNAi therapies remain experimental for IBD; leaves open confirmation of preclinical efficacy in large trials.
Antisense oligonucleotide (ASO) technology is a specific, rapid, and potentially high-throughput method for inhibiting gene expression. ASO refers to a short synthetic strand of deoxy-ribonucleotide analogue that hybridizes with the complementary mRNA via Watson–Crick base pairing. The resulting RNA-DNA heteroduplex leads to a reduced translation of the target transcript thus inducing a blockade in the transfer of genetic information from DNA to protein. Crohn's disease (CD) and ulcerative colitis (UC), the major inflammatory bowel diseases (IBD) in humans, are clinically characterized by intermittent exacerbations and sometimes chronically active course. The causes of CD and UC are unknown and there is not a curative therapy for these patients. However, in the last decades, the advent of new techniques of molecular biology has largely advanced our understanding of the mechanisms that drive tissue damage in IBD. These progresses have facilitated the development of new therapeutic compounds, which selectively target molecules involved in IBD pathogenesis. This short review provides a comprehensive understanding of the basic concepts underlying the mechanism of action of ASOs and summarizes the available pre-clinical and clinical data for ASO-based therapy in IBD.
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Fusco et al. (2019) conducted a review in Inflammatory Bowel Disease (Crohn's disease and ulcerative colitis). Antisense Oligonucleotides (ASOs) and RNA interference (RNAi) was evaluated. Antisense oligonucleotides and RNA interference represent promising therapeutic strategies for inflammatory bowel disease, though large clinical trials have yet to confirm the efficacy seen in preclinical models.
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