Naeem Sajid, Zhang Ju, Zhang Yang, Wang Yu
College of Life Sciences and Oceanography, Shenzhen University, Shenzhen 518060, China.
College of Physics and Optoelectronic Engineering, Shenzhen University, Shenzhen 518060, China.
Mol Ther Nucleic Acids. 2024 Dec 23;36(1):102440. doi: 10.1016/j.omtn.2024.102440. eCollection 2025 Mar 11.
Nucleic acid therapeutics have become increasingly recognized in recent years for their capability to target both coding and non-coding sequences. Several types of nucleic acid modalities, including siRNA, mRNA, aptamer, along with antisense oligo, have been approved by regulatory bodies for therapeutic use. The field of nucleic acid therapeutics has been brought to the forefront by the rapid development of vaccines against COVID-19, followed by a number of approvals for clinical use including much anticipated CRISPR-Cas9. However, obstacles such as the difficulty of achieving efficient and targeted delivery to diseased sites remain. This review provides an overview of nucleic acid therapeutics and highlights substantial advancements, including critical engineering, conjugation, and delivery strategies, that are paving the way for their growing role in modern medicine.
近年来,核酸疗法因其能够靶向编码和非编码序列的能力而越来越受到认可。几种类型的核酸形式,包括小干扰RNA(siRNA)、信使核糖核酸(mRNA)、适体以及反义寡核苷酸,已获得监管机构批准用于治疗。针对2019冠状病毒病(COVID-19)疫苗的迅速发展将核酸治疗领域推到了前沿,随后有多项临床使用批准,包括备受期待的CRISPR-Cas9。然而,诸如难以实现高效且靶向递送至患病部位等障碍仍然存在。本综述概述了核酸疗法,并强调了包括关键工程、缀合和递送策略在内的重大进展,这些进展正在为其在现代医学中日益重要的作用铺平道路。