Antisense technology: A review

被引：151
作者：
Crooke, Stanley T. [1 ]
Liang, Xue-Hai [1 ]
Baker, Brenda F. [2 ]
Crooke, Rosanne M. [3 ]
机构：
[1] Ionis Pharmaceut Inc, Core Antisense Res, Carlsbad, CA 92008 USA
[2] Ionis Pharmaceut Inc, Dev Commun, Carlsbad, CA USA
[3] Ionis Pharmaceut Inc, Antisense Drug Discovery, Carlsbad, CA USA
来源：
JOURNAL OF BIOLOGICAL CHEMISTRY | 2021年 / 296卷
关键词：
HUMAN RNASE H1; DUCHENNE MUSCULAR-DYSTROPHY; RESISTANT PROSTATE-CANCER; I DOSE-ESCALATION; PHOSPHOROTHIOATE OLIGONUCLEOTIDES; MESSENGER-RNA; DOUBLE-BLIND; PHASE-I; TARGETING APOLIPOPROTEIN(A); HETERODUPLEX SUBSTRATE;
D O I：
10.1016/j.jbc.2021.100416
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
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.
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页数：39
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