Receptors, endocytosis, and trafficking: the biological basis of targeted delivery of antisense and siRNA oligonucleotides

J Drug Target. 2013 Jan;21(1):27-43. doi: 10.3109/1061186X.2012.740674. Epub 2012 Nov 19.

Abstract

The problem of targeted delivery of antisense and siRNA oligonucleotides can be resolved into two distinct aspects. The first concerns devising ligand-oligonucleotide or ligand-carrier moieties that bind with high selectivity to receptors on the cell type of interest and that are efficiently internalized by endocytosis. The second concerns releasing oligonucleotides from pharmacologically inert endomembrane compartments so that they can access RNA in the cytosol or nucleus. In this review, we will address both of these aspects. Thus, we present information on three important receptor families, the integrins, the receptor tyrosine kinases, and the G protein-coupled receptors in terms of their suitability for targeted delivery of oligonucleotides. This includes discussion of receptor abundance, internalization and trafficking pathways, and the availability of suitable high affinity ligands. We also consider the process of oligonucleotide uptake and intracellular trafficking and discuss approaches to modulating these processes in a pharmacologically productive manner. Hopefully, the basic information presented in this review will be of value to investigators involved in designing delivery approaches for oligonucleotides.

Publication types

  • Review

MeSH terms

  • Animals
  • Cell Nucleus / metabolism
  • Cytosol / metabolism
  • Endocytosis
  • Humans
  • Integrins / metabolism
  • Oligonucleotides, Antisense / administration & dosage*
  • RNA / metabolism*
  • RNA, Small Interfering / administration & dosage*
  • Receptor Protein-Tyrosine Kinases / metabolism
  • Receptors, G-Protein-Coupled / metabolism

Substances

  • Integrins
  • Oligonucleotides, Antisense
  • RNA, Small Interfering
  • Receptors, G-Protein-Coupled
  • RNA
  • Receptor Protein-Tyrosine Kinases