Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation.

IF 4 2区医学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY

Nucleic acid therapeutics Pub Date : 2024-04-01 Epub Date: 2024-03-20 DOI:10.1089/nat.2023.0068

Jillian Belgrad, Hassan H Fakih, Anastasia Khvorova

引用次数: 0

Abstract

Nucleic acid-based therapies have become the third major drug class after small molecules and antibodies. The role of nucleic acid-based therapies has been strengthened by recent regulatory approvals and tremendous clinical success. In this review, we look at the major obstacles that have hindered the field, the historical milestones that have been achieved, and what is yet to be resolved and anticipated soon. This review provides a view of the key innovations that are expanding nucleic acid capabilities, setting the stage for the future of nucleic acid therapeutics.

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核酸疗法：成功、里程碑和即将到来的创新。

核酸疗法已成为继小分子和抗体之后的第三大药物类别。最近获得的监管批准和巨大的临床成功加强了核酸疗法的作用。在这篇综述中，我们将探讨阻碍这一领域发展的主要障碍、已取得的历史性里程碑，以及哪些问题尚待解决并有望很快解决。本综述介绍了正在扩展核酸能力的关键创新，为核酸疗法的未来奠定了基础。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Nucleic acid therapeutics BIOCHEMISTRY & MOLECULAR BIOLOGY-CHEMISTRY, MEDICINAL

CiteScore

7.60

自引率

7.50%

发文量

审稿时长

>12 weeks

期刊介绍： Nucleic Acid Therapeutics is the leading journal in its field focusing on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. The Journal examines many new approaches for using nucleic acids as therapeutic agents or in modifying nucleic acids for therapeutic purposes including: oligonucleotides, gene modification, aptamers, RNA nanoparticles, and ribozymes.