Ikerbasque, Basque Foundation for Science, Bilbao, Spain.
Neuromuscular Disorders Research Group, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain.
Methods Mol Biol. 2022;2434:33-49. doi: 10.1007/978-1-0716-2010-6_2.
Nucleic acid therapeutics is a growing field aiming to treat human conditions that has gained special attention due to the successful development of mRNA vaccines against SARS-CoV-2. Another type of nucleic acid therapeutics is antisense oligonucleotides, versatile tools that can be used in multiple ways to target pre-mRNA and mRNA. While some years ago these molecules were just considered a useful research tool and a curiosity in the clinical market, this has rapidly changed. These molecules are promising strategies for personalized treatments for rare genetic diseases and they are in development for very common disorders too. In this chapter, we provide a brief description of the different mechanisms of action of these RNA therapeutic molecules, with clear examples at preclinical and clinical stages.
核酸疗法是一个不断发展的领域，旨在治疗人类疾病，由于针对 SARS-CoV-2 的 mRNA 疫苗的成功开发，该领域受到了特别关注。另一种核酸疗法是反义寡核苷酸，这是一种多功能工具，可通过多种方式靶向 pre-mRNA 和 mRNA。虽然几年前，这些分子还只是被认为是一种有用的研究工具和临床市场上的一种新奇事物，但这种情况已经迅速发生了变化。这些分子是治疗罕见遗传疾病的个性化治疗的有前途的策略，它们也正在开发用于非常常见的疾病。在本章中，我们简要描述了这些 RNA 治疗分子的不同作用机制，并提供了临床前和临床阶段的明确示例。
