Reichel Liên S, Traeger Anja
Institute of Organic and Macromolecular Chemistry (IOMC), Friedrich Schiller University Jena, Jena, Germany.
Jena Center for Soft Matter (JCSM), Friedrich Schiller University Jena, Jena, Germany.
Handb Exp Pharmacol. 2024;284:27-43. doi: 10.1007/164_2023_694.
Considering nucleic acids as the language of life and the genome as the instruction manual of cells, their targeted modulation promises great opportunities in treating and healing diseases. In addition to viral gene transfer, the overwhelming power of non-viral mRNA-based vaccines is driving the development of novel gene transporters. Thereby, various nucleic acids such as DNA (pDNA) or RNA (mRNA, siRNA, miRNA, gRNA, or ASOs) need to be delivered, requiring a transporter due to their high molar mass and negative charge in contrast to classical agents. This chapter presents the specific biological hurdles for using nucleic acids and shows how new materials can overcome these.
将核酸视为生命的语言，基因组视为细胞的说明书，对它们进行靶向调控有望为疾病治疗和治愈带来巨大机遇。除了病毒基因传递外，基于非病毒信使核糖核酸的疫苗的强大作用正在推动新型基因转运体的发展。因此，需要递送各种核酸，如脱氧核糖核酸（pDNA）或核糖核酸（mRNA、小干扰核糖核酸、微小核糖核酸、引导核糖核酸或反义寡核苷酸），与传统药物相比，由于它们的高摩尔质量和负电荷，需要一种转运体。本章介绍了使用核酸时面临的具体生物学障碍，并展示了新材料如何克服这些障碍。
