Key Laboratory of Sensing Technology and Biomedical Instrument of Guangdong Province, School of Biomedical Engineering, Sun Yat-sen University, Guangzhou, 510006, Guangdong Province, China.
J Mater Chem B. 2020 Jul 14;8(26):5564-5577. doi: 10.1039/d0tb00498g. Epub 2020 Jun 4.
Gene therapy is deemed to be a powerful and promising tool to treat many stubborn diseases in the near future. For successful gene therapy, gene vectors play a vital role as they determine whether the edited gene can be loaded and transferred into target cells effectively. However, traditional gene vectors face some serious challenges, especially in biosafety and trans-membrane efficiency. Recently, inspired by cell-penetrating peptides (CPPs) possessing excellent biosafety and trans-membrane efficiency, various types of arginine-based gene delivery systems have been designed and fabricated. In this review, the superiority and mechanism of action of arginine in promoting membrane penetration are briefly presented. Then, four types of arginine-based gene vectors, namely, typical linear polymers, dendrimers, lipids and arginine-based composite gene vectors and their applications are discussed in detail. Finally, the current challenges and future perspectives of arginine-based gene delivery systems are discussed.
基因治疗被认为是一种强大且有前途的工具，可以在不久的将来治疗许多顽固疾病。对于成功的基因治疗，基因载体起着至关重要的作用，因为它们决定了编辑后的基因是否能够有效地被加载和转移到靶细胞中。然而，传统的基因载体面临一些严重的挑战，特别是在生物安全性和跨膜效率方面。最近，受具有优异生物安全性和跨膜效率的细胞穿透肽（CPP）的启发，已经设计和制造了各种类型的基于精氨酸的基因传递系统。在这篇综述中，简要介绍了精氨酸促进膜穿透的优越性和作用机制。然后，详细讨论了四种基于精氨酸的基因载体，即典型的线性聚合物、树枝状聚合物、脂质和基于精氨酸的复合基因载体及其应用。最后，讨论了基于精氨酸的基因传递系统的当前挑战和未来展望。
