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Open AccessReview

Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

Department of Pharmaceutical Science, University of Connecticut, Storrs, CT 06269, USA
Department of Genetics, Yale University, New Haven, CT 06520, USA
Department of Radiation Oncology, Vanderbilt University Medical Center, Nashville, TN 37232-5671, USA
Department of Chemistry, Wesleyan University, Middletown, CT 06459, USA
Division of Cardiovascular Medicine, Department of Internal Medicine, The University of Iowa, Iowa City, IA 52242, USA
Author to whom correspondence should be addressed.
J. Clin. Med. 2020, 9(6), 2004;
Received: 24 April 2020 / Revised: 20 June 2020 / Accepted: 24 June 2020 / Published: 26 June 2020
(This article belongs to the Section Pharmacology)
Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and modulate protein expression through several different mechanisms. The ASO field is an emerging area of drug development that targets the disease source at the RNA level and offers a promising alternative to therapies targeting downstream processes. To translate ASO-based therapies into a clinical success, it is crucial to overcome the challenges associated with off-target side effects and insufficient biological activity. In this regard, several chemical modifications and diverse delivery strategies have been explored. In this review, we systematically discuss the chemical modifications, mechanism of action, and optimized delivery strategies of several different classes of ASOs. Further, we highlight the recent advances made in development of ASO-based drugs with a focus on drugs that are approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for clinical applications. We also discuss various promising ASO-based drug candidates in the clinical trials, and the outstanding opportunity of emerging microRNA as a viable therapeutic target for future ASO-based therapies. View Full-Text
Keywords: RNA; antisense oligonucleotides; chemical modifications; clinical trials RNA; antisense oligonucleotides; chemical modifications; clinical trials
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Dhuri, K.; Bechtold, C.; Quijano, E.; Pham, H.; Gupta, A.; Vikram, A.; Bahal, R. Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development. J. Clin. Med. 2020, 9, 2004.

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