Overcoming Pharmaceutical Bottlenecks for Nucleic Acid Drug Development,Accounts of Chemical Research

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Overcoming Pharmaceutical Bottlenecks for Nucleic Acid Drug Development
Accounts of Chemical Research ( IF 16.4 ) Pub Date : 2023-01-09 , DOI: 10.1021/acs.accounts.2c00464
Mei Lu _{1,

2} , Haonan Xing _{2,

3} , Aiping Zheng ₃ , Yuanyu Huang ₁ , Xing-Jie Liang _{1,

2}

Affiliation

The outbreak of the coronavirus disease 2019 (COVID-19) pandemic and swift approval of two mRNA vaccines have put nucleic acid therapeutics in the spotlight of both the scientific community and the general public. Actually, in addition to mRNAs, multiple nucleic acid therapeutics have been successively commercialized over the past few years. The rapid development of nucleic acid drugs not only demonstrates their superior potency but also marks a new era of the field. Compared with conventional treatments targeting proteins rather than the root causes of diseases at the genetic level, nucleic acids are capable of achieving long-standing or even curative effects against undruggable disorders by modulating gene expression via inhibition, editing, addition, or replacement. This offers a terrific arsenal for expanding therapeutic access to diseases lacking current treatment options and developing vaccines to provide swift responses to emerging global health threats.

中文翻译：

克服核酸药物研发的制药瓶颈

2019 年冠状病毒病 (COVID-19) 大流行的爆发和两种 mRNA 疫苗的迅速批准使核酸疗法成为科学界和公众关注的焦点。事实上，除了mRNAs，在过去的几年里，多种核酸药物也相继实现了商业化。核酸药物的快速发展，不仅展示了其优越的药效，也标志着该领域进入了一个新时代。与针对蛋白质而不是基因水平疾病根源的常规治疗相比，核酸能够通过抑制、编辑、添加或替换调节基因表达，从而对不可药性的疾病产生长期甚至治愈的效果。

更新日期：2023-01-09

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