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Oligonucleotides to the (Gene) Rescue: FDA Approvals 2017-2019.
Trends in Pharmacological Sciences ( IF 13.9 ) Pub Date : 2019-12-10 , DOI: 10.1016/j.tips.2019.10.009
Jacqueline Rüger 1 , Silvia Ioannou 2 , Daniela Castanotto 1 , Cy A Stein 3
Affiliation  

Four decades have passed since oligonucleotides were first used to manipulate gene expression. There were few FDA approvals prior to 2016, mostly of drugs that eventually exhibited poor performance in the market. The aura of their younger siRNA relatives had also faded during the past 15 years. However, several FDA approvals have occurred in the past 4 years, restoring hope that a new era has dawned in oligonucleotide/siRNA clinical therapeutics. Here, we review the field of oligonucleotide therapeutics and provide an update on FDA approvals of oligonucleotides from 2017 until the second quarter of 2019. We take into consideration the ethical issues looming over the still somewhat limited efficacy of these molecules, the toxicity of treatment, and the exorbitant cost of these therapeutic agents, which limits accessibility for many.

中文翻译:

(基因)救援的寡核苷酸:2017-2019年FDA批准。

自从首次将寡核苷酸用于操纵基因表达以来,已经过去了40年。2016年之前很少有FDA批准,大多数最终在市场上表现不佳的药物。在过去的15年中,年轻的siRNA亲戚的光环也逐渐消失。但是,在过去的4年中,已经获得了FDA的多项批准,这重燃了人们对寡核苷酸/ siRNA临床治疗学新时代的希望。在这里,我们回顾了寡核苷酸治疗领域,并提供了从2017年到2019年第二季度FDA寡核苷酸批准的最新信息。我们考虑到这些分子的疗效仍然有限的伦理问题,治疗的毒性,以及这些治疗剂的高昂成本,这限制了许多人的可及性。
更新日期:2019-12-11
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