Delivery of RNA interference therapeutics using polycation-based nanoparticles

Adv Drug Deliv Rev. 2009 Jul 25;61(9):710-20. doi: 10.1016/j.addr.2009.04.001. Epub 2009 Apr 5.

Abstract

RNAi-based therapies are dependent on extracellular and intracellular delivery of RNA molecules for enabling target interaction. Polycation-based nanoparticles (or polyplexes) formed by self-assembly with RNA can be used to modulate pharmacokinetics and intracellular trafficking to improve the therapeutic efficacy of RNAi-based therapeutics. This review describes the application of polyplexes for extracellular and intracellular delivery of synthetic RNA molecules. Focus is given to routes of administration and silencing effects in animal disease models. The inclusion of functional components into the nanoparticle for controlling cellular trafficking and RNA release is discussed. This work highlights the versatile nature of polycation-based nanoparticles to fulfil the delivery requirements for RNA molecules with flexibility in design to evolve alongside an expanding repertoire of RNAi-based drugs.

Publication types

  • Review

MeSH terms

  • Animals
  • Biological Transport
  • Gene Silencing
  • Humans
  • Injections
  • Nanoparticles / administration & dosage*
  • Polyamines / administration & dosage*
  • Polyelectrolytes
  • Polyethylene Glycols / administration & dosage
  • RNA Interference*
  • RNA, Small Interfering / administration & dosage*

Substances

  • Polyamines
  • Polyelectrolytes
  • RNA, Small Interfering
  • polycations
  • Polyethylene Glycols