Non-viral nucleic acid delivery methods

Expert Opin Biol Ther. 2017 Jan;17(1):105-118. doi: 10.1080/14712598.2017.1248941. Epub 2016 Nov 9.

Abstract

Delivery of nucleic acid-based molecules in human cells is a highly studied approach for the treatment of several disorders including monogenic diseases and cancers. Non-viral vectors for DNA and RNA transfer, although in general less efficient than virus-based systems, are particularly well adapted mostly due to the absence of biosafety concerns. Non-viral methods could be classified in two main groups: physical and vector-assisted delivery systems. Both groups comprise several different methods, none of them universally applicable. The choice of the optimal method depends on the predefined objectives and the features of targeted micro-environment. Areas covered: In this review, the authors discuss non-viral techniques and present recent therapeutic achievements in ex vivo and in vivo nucleic acid delivery by most commonly used techniques while emphasizing the role of 'biological particles', namely peptide transduction domains, virus like particles, gesicles and exosomes. Expert opinion: The number of available non-viral transfection techniques used for human therapy increased rapidly, followed by still moderate success in efficacy. The prospects are to be found in design of multifunctional hybrid systems that reflect the viral efficiency. In this respect, biological particles are very promising.

Keywords: Nucleic acid delivery; exosomes; particle mediated transfection; peptide transduction domains; physical transfection; virus derived particles.

Publication types

  • Review

MeSH terms

  • Animals
  • DNA / administration & dosage
  • DNA / genetics
  • Gene Transfer Techniques*
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage*
  • Genetic Vectors / genetics
  • Humans
  • Nucleic Acids / administration & dosage*
  • Nucleic Acids / genetics
  • Transfection / methods

Substances

  • Nucleic Acids
  • DNA