Antisense technology: A review.

Chemistry, Pharmaceutical Clinical Trials as Topic Drug Discovery Humans Oligonucleotides, Antisense / pharmacology RNA, Small Interfering / pharmacology
RNase H1 antisense clinical results molecular mechanisms splicing

Journal

The Journal of biological chemistry
ISSN: 1083-351X
Titre abrégé: J Biol Chem
Pays: United States
ID NLM: 2985121R

Informations de publication

Date de publication:
Historique:
received: 25 09 2020
revised: 09 02 2021
accepted: 10 02 2021
pubmed: 19 2 2021
medline: 31 7 2021
entrez: 18 2 2021
Statut: ppublish

Résumé

Antisense technology is beginning to deliver on the broad promise of the technology. Ten RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double-strand ASOs (siRNAs) have now been approved for commercial use, and the ASOs in phase 2/3 trials are innovative, delivered by multiple routes of administration and focused on both rare and common diseases. In fact, two ASOs are used in cardiovascular outcome studies and several others in very large trials. Interest in the technology continues to grow, and the field has been subject to a significant number of reviews. In this review, we focus on the molecular events that result in the effects observed and use recent clinical results involving several different ASOs to exemplify specific molecular mechanisms and specific issues. We conclude with the prospective on the technology.

Identifiants

DOI: 10.1016/j.jbc.2021.100416 PMID: 33600796 PMC: PMC8005817
pubmed: 33600796
pii: S0021-9258(21)00189-7
doi: 10.1016/j.jbc.2021.100416
pmc: PMC8005817
pii:
doi:

Substances chimiques

Oligonucleotides, Antisense 0
RNA, Small Interfering 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

100416

Informations de copyright

Copyright © 2021 The Authors. Published by Elsevier Inc. All rights reserved.

Déclaration de conflit d'intérêts

Conflict of interest The authors declare that they have no conflicts of interest with the contents of this article.

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Auteurs

Stanley T Crooke (ST)

Core Antisense Research, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA. Electronic address: scrooke@ionisph.com.

Xue-Hai Liang (XH)

Core Antisense Research, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA.

Brenda F Baker (BF)

Development Communication, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA.

Rosanne M Crooke (RM)

Antisense Drug Discovery, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA.

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Classifications MeSH

questionsmedicales.fr › Disciplines des sciences naturelles › Chimie › Chimie pharmaceutique › Antisense technology: A review.
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Antisense technology: A review.
questionsmedicales.fr › Techniques d'investigation › Développement de médicament › Découverte de médicament › Antisense technology: A review.
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Antisense technology: A review.
questionsmedicales.fr › Actions chimiques et utilisations › Utilisations spécialisées de produits chimiques › Produits chimiques de laboratoire › Sondes moléculaires › Sondes d'acide nucléique › Éléments antisens (génétique) › Oligonucléotides antisens › Antisense technology: A review.
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Acides nucléiques › ARN › ARN non traduit › Petit ARN non traduit › Petit ARN interférent › Antisense technology: A review.