Cellular uptake and intracellular trafficking of antisense and siRNA oligonucleotides

Bioconjug Chem. 2012 Feb 15;23(2):147-57. doi: 10.1021/bc200377d. Epub 2011 Oct 27.

Abstract

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.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Endocytosis*
  • Humans
  • Intracellular Space / metabolism*
  • Oligonucleotides / metabolism*
  • Oligonucleotides / therapeutic use
  • RNA, Small Interfering / metabolism*
  • RNA, Small Interfering / therapeutic use

Substances

  • Oligonucleotides
  • RNA, Small Interfering