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Antisense technology: A review

The Journal of biological chemistry, 2021-01, Vol.296, p.100416-100416, Article 100416 [Peer Reviewed Journal]

2021 The Authors ;Copyright © 2021 The Authors. Published by Elsevier Inc. All rights reserved. ;2021 The Authors 2021 ;ISSN: 0021-9258 ;EISSN: 1083-351X ;DOI: 10.1016/j.jbc.2021.100416 ;PMID: 33600796

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  • Title:
    Antisense technology: A review
  • Author: Crooke, Stanley T. ; Liang, Xue-Hai ; Baker, Brenda F. ; Crooke, Rosanne M.
  • Subjects: antisense ; clinical results ; JBC Reviews ; molecular mechanisms ; RNase H1 ; splicing
  • Is Part Of: The Journal of biological chemistry, 2021-01, Vol.296, p.100416-100416, Article 100416
  • Description: 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.
  • Publisher: United States: Elsevier Inc
  • Language: English
  • Identifier: ISSN: 0021-9258
    EISSN: 1083-351X
    DOI: 10.1016/j.jbc.2021.100416
    PMID: 33600796
  • Source: Open Access: PubMed Central
    Directory of Open Access Journals
    Alma/SFX Local Collection
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