Division of Molecular Pharmaceutics, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, North Carolina 27599, USA.
Bioconjug Chem. 2012 Feb 15;23(2):147-57. doi: 10.1021/bc200377d. Epub 2011 Oct 27.
Significant progress is being made concerning the development of oligonucleotides as therapeutic agents. Studies with antisense, siRNA, and other forms of oligonucleotides have shown promise in cellular and animal models and in some clinical studies. Nonetheless, our understanding of how oligonucleotides function in cells and tissues is really quite limited. One major issue concerns the modes of uptake and intracellular trafficking of oligonucleotides, whether as "free" molecules or linked to various delivery moieties such as nanoparticles or targeting ligands. In this review, we examine the recent literature on oligonucleotide internalization and subcellular trafficking in the context of current insights into the basic machinery for endocytosis and intracellular vesicular traffic.
在将寡核苷酸开发为治疗剂方面正在取得重大进展。反义寡核苷酸、siRNA 和其他形式的寡核苷酸在细胞和动物模型以及一些临床研究中显示出了前景。尽管如此,我们对寡核苷酸在细胞和组织中的作用机制的理解还相当有限。一个主要问题涉及寡核苷酸的摄取和细胞内转运方式,无论是作为“游离”分子还是与各种递药部分(如纳米颗粒或靶向配体)相连。在这篇综述中,我们根据目前对内吞作用和细胞内囊泡运输基本机制的了解,检查了有关寡核苷酸内化和亚细胞转运的最新文献。
Bioconjug Chem. 2011-10-27
Expert Opin Drug Deliv. 2011-3-8
Adv Drug Deliv Rev. 2015-6-29
Nucleic Acids Res. 2008-7
Nucleic Acids Res. 2016-8-19
Acc Chem Res. 2019-3-26
Nucleic Acid Ther. 2018-4-30
Angew Chem Int Ed Engl. 2019-4-5
Mol Ther Nucleic Acids. 2025-5-24
J Pharmacokinet Pharmacodyn. 2025-6-12
CPT Pharmacometrics Syst Pharmacol. 2024-5
Mol Ther Nucleic Acids. 2023-8-18
Expert Opin Drug Deliv. 2011-3-8
Nucleic Acids Res. 2011-2-23
Nucleic Acids Res. 2011-2-23