Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

Karishma Dhuri; Clara Bechtold; Elias Quijano; Ha Pham; Anisha Gupta; Ajit Vikram; Raman Bahal

doi:10.3390/jcm9062004

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Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

Journal article

Open access

Peer reviewed

Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

Karishma Dhuri, Clara Bechtold, Elias Quijano, Ha Pham, Anisha Gupta, Ajit Vikram and Raman Bahal

Journal of clinical medicine, Vol.9(6), p.2004

06/26/2020

DOI: 10.3390/jcm9062004

PMCID: PMC7355792

PMID: 32604776

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Abstract

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.

antisense oligonucleotides

chemical modifications

clinical trials

Review

RNA

Details

Title: Subtitle: Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development
Creators: Karishma Dhuri - University of Connecticut
Clara Bechtold - University of Connecticut
Elias Quijano - Yale University
Ha Pham - Vanderbilt University Medical Center
Anisha Gupta - Wesleyan University
Ajit Vikram - University of Iowa
Raman Bahal - University of Connecticut
Resource Type: Journal article
Publication Details: Journal of clinical medicine, Vol.9(6), p.2004
DOI: 10.3390/jcm9062004
PMID: 32604776
PMCID: PMC7355792
NLM abbreviation: J Clin Med
ISSN: 2077-0383
eISSN: 2077-0383
Publisher: MDPI
Grant note: DOI: 10.13039/100000009, name: National Institutes of Health, award: 1R01HL147028-01A1
Language: English
Date published: 06/26/2020
Academic Unit: Cardiovascular Medicine; Fraternal Order of Eagles Diabetes Research Center; Internal Medicine
Record Identifier: 9984359928502771

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