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Therapeutic Oligonucleotides for Patients with Inflammatory Bowel Diseases

Authors Marafini I, Monteleone G

Received 16 April 2020

Accepted for publication 30 May 2020

Published 15 June 2020 Volume 2020:14 Pages 47—51


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Dr Doris Benbrook

Irene Marafini, Giovanni Monteleone

Department of Systems Medicine, University of Rome “Tor Vergata”, Rome, Italy

Correspondence: Giovanni Monteleone
Dipartimento di Medicina dei Sistemi, Università di Roma “Tor Vergata”, via Montpellier, 1, Rome 00133, Italy
Tel +390620903702
Fax +390672596391

Introduction: 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.
Areas Covered: In this short review, we summarize experimental and clinical data on the use of ASOs in IBD.
Expert Opinion: 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.

Keywords: Crohn’s disease, ulcerative colitis, ICAM-1, Smad7

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