Therapeutic Oligonucleotides for Patients with Inflammatory Bowel Diseases

The better understanding of the molecular mechanisms, which drive the pathological process in the gut of patients with Crohn's disease (CD) and patients with ulcerative colitis (UC), the major forms of inflammatory bowel diseases (IBD) in humans, has facilitated the development of novel therapeutic compounds. Among these, antisense oligonucleotides (ASOs) have been used to inhibit the expression of molecules, which sustain the IBD-associated mucosal inflammation.

In this short review, we summarize experimental and clinical data on the use of ASOs in IBD.

Preclinical work indicates that the modulation of specific inflammatory pathways through the use of ASOs is highly effective and associates with low risk of adverse events. Initial clinical studies have confirmed the benefit of some ASOs even though no compound has yet reached the market. Further experimentation is warranted to establish the optimal route of administration for each ASO, ascertain whether and how long ASOs maintain their activity following administration, and identify which patient can benefit from specific ASO treatment.