Efficient cell penetration and delivery of peptide nucleic acids by an argininocalix[4]arene

Sci Rep. 2019 Feb 28;9(1):3036. doi: 10.1038/s41598-019-39211-4.

Abstract

The application of Peptide Nucleic Acids (PNAs), mimics of DNA lacking the sugar-phosphate backbone, for antisense/anti-gene therapy and gene editing is limited by their low uptake by cells. Currently, no simple and efficient delivery systems and methods are available to solve this open issue. One of the most promising approach is the modification of the PNA structure through the covalent linkage of poliarginine tails, but this means that every PNA intended to be internalized must be modified. Herein we report the results relative to the delivery ability of a macrocyclic multivalent tetraargininocalix[4]arene (1) used as non-covalent vector for anti-miR-221-3p PNAs. High delivery efficiency, low cytotoxicity, maintenance of the PNA biological activity and ease preparation of the transfection formulation, simply attained by mixing PNA and calixarene, candidate this vector as universal delivery system for this class of nucleic acid analogues.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line, Tumor
  • DNA / metabolism
  • Humans
  • MicroRNAs / metabolism
  • Peptide Nucleic Acids / metabolism*
  • Transfection / methods

Substances

  • MicroRNAs
  • Peptide Nucleic Acids
  • DNA