Challenges and Opportunities for Nucleic Acid Therapeutics

被引：44

作者：

Corey, David R. ^{[1
]}

Damha, Masad J. ^{[2
]}

Manoharan, Muthiah ^{[3
]}

机构：

[1] UT Southwestern Med Ctr, Dept Pharmacol & Biochem, 6001 Forest Pk Rd, Dallas, TX 75390 USA

[2] McGill Univ, Dept Chem, Montreal, PQ, Canada

[3] Alnylam Pharmaceut, Cambridge, MA USA

来源：

NUCLEIC ACID THERAPEUTICS | 2022年 / 32卷 / 01期

基金：

美国国家卫生研究院;

关键词：

antisense; siRNA; therapeutic; ANTISENSE OLIGONUCLEOTIDES; GENE; RNA; DELIVERY; HEPATOCYTES;

D O I：

10.1089/nat.2021.0085

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：

After decades overcoming difficult problems, antisense oligonucleotide (ASO), duplex RNA (siRNA), and messenger RNA (mRNA) nucleic acid therapeutic strategies are finally demonstrating clinical benefits. This success presents new challenges. What goals remain for basic research Will there be an explosion of clinical applications that benefit many patients with different diseases, or will success be restricted to diseases that are ideal for the application of current technologies The aim of this perspective is to describe a selection of the major goals for the next decade. © Copyright 2022, Mary Ann Liebert, Inc., publishers 2022.

引用

页码：8 / 13

页数：6

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