Antisense technology: A review

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 b...

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Bibliographic Details
Published inThe Journal of biological chemistry Vol. 296; p. 100416
Main Authors Crooke, Stanley T., Liang, Xue-Hai, Baker, Brenda F., Crooke, Rosanne M.
Format Journal Article
LanguageEnglish
Published United States Elsevier Inc 01.01.2021
American Society for Biochemistry and Molecular Biology
Subjects
antisense
clinical results
JBC Reviews
molecular mechanisms
RNase H1
splicing
RTD
SGLT2
clinical results
ds
GalNAc
TIE
molecular mechanisms
GFR
PD
FCS
splicing
PK
SMN1
ss ASO
RNase H1
antisense
Online AccessGet full text
ISSN0021-9258
1083-351X
1083-351X
DOI10.1016/j.jbc.2021.100416

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Abstract 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.
AbstractList 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.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.
Antisense technology is beginning to deliver on the broad promise of the technology. Ten RNA Targeted drugs including 8 single strand antisense drugs (ASOs) and 2 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 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.
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.
ArticleNumber 100416
Author Crooke, Stanley T.
Baker, Brenda F.
Crooke, Rosanne M.
Liang, Xue-Hai
Author_xml – sequence: 1
  givenname: Stanley T.
  orcidid: 0000-0002-4260-1520
  surname: Crooke
  fullname: Crooke, Stanley T.
  email: scrooke@ionisph.com
  organization: Core Antisense Research, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA
– sequence: 2
  givenname: Xue-Hai
  orcidid: 0000-0001-9333-8622
  surname: Liang
  fullname: Liang, Xue-Hai
  organization: Core Antisense Research, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA
– sequence: 3
  givenname: Brenda F.
  orcidid: 0000-0001-8586-768X
  surname: Baker
  fullname: Baker, Brenda F.
  organization: Development Communication, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA
– sequence: 4
  givenname: Rosanne M.
  surname: Crooke
  fullname: Crooke, Rosanne M.
  organization: Antisense Drug Discovery, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA
BackLink https://www.ncbi.nlm.nih.gov/pubmed/33600796$$D View this record in MEDLINE/PubMed
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10.1093/nar/gkv964
10.1038/nm0696-668
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Thu Apr 24 23:05:32 EDT 2025
Fri Feb 23 02:43:02 EST 2024
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Keywords RTD
SGLT2
clinical results
ds
GalNAc
TIE
molecular mechanisms
GFR
PD
FCS
splicing
PK
SMN1
ss ASO
RNase H1
antisense
Language English
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Snippet Antisense technology is beginning to deliver on the broad promise of the technology. Ten RNA-targeted drugs including eight single-strand antisense drugs...
Antisense technology is beginning to deliver on the broad promise of the technology. Ten RNA Targeted drugs including 8 single strand antisense drugs (ASOs)...
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StartPage 100416
SubjectTerms antisense
clinical results
JBC Reviews
molecular mechanisms
RNase H1
splicing
Title Antisense technology: A review
URI https://dx.doi.org/10.1016/j.jbc.2021.100416
https://www.ncbi.nlm.nih.gov/pubmed/33600796
https://www.proquest.com/docview/2491952502
https://pubmed.ncbi.nlm.nih.gov/PMC8005817
Volume 296
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