Therapeutic applications of extracellular vesicles: clinical promise and open questions

Annu Rev Pharmacol Toxicol. 2015;55:439-464. doi: 10.1146/annurev-pharmtox-010814-124630. Epub 2014 Oct 3.


This review provides an updated perspective on rapidly proliferating efforts to harness extracellular vesicles (EVs) for therapeutic applications. We summarize current knowledge, emerging strategies, and open questions pertaining to clinical potential and translation. Potentially useful EVs comprise diverse products of various cell types and species. EV components may also be combined with liposomes and nanoparticles to facilitate manufacturing as well as product safety and evaluation. Potential therapeutic cargoes include RNA, proteins, and drugs. Strategic issues considered herein include choice of therapeutic agent, means of loading cargoes into EVs, promotion of EV stability, tissue targeting, and functional delivery of cargo to recipient cells. Some applications may harness natural EV properties, such as immune modulation, regeneration promotion, and pathogen suppression. These properties can be enhanced or customized to enable a wide range of therapeutic applications, including vaccination, improvement of pregnancy outcome, and treatment of autoimmune disease, cancer, and tissue injury.

Keywords: drug delivery; exosomes; extracellular RNA; gene medicine; gene therapy; liposomes; mesenchymal stem cells; microvesicles; nanoparticles.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Cell-Derived Microparticles / immunology
  • Cell-Derived Microparticles / metabolism*
  • Chemistry, Pharmaceutical
  • DNA / administration & dosage*
  • DNA / metabolism
  • Drug Carriers*
  • Drug Design*
  • Exosomes / immunology
  • Exosomes / metabolism*
  • Gene Transfer Techniques*
  • Humans
  • Nanoparticles
  • Nanotechnology
  • Pharmaceutical Preparations / administration & dosage*
  • Pharmaceutical Preparations / metabolism
  • RNA / administration & dosage*
  • RNA / metabolism
  • Tissue Distribution
  • Vaccines / administration & dosage*
  • Vaccines / immunology
  • Vaccines / pharmacokinetics


  • Drug Carriers
  • Pharmaceutical Preparations
  • Vaccines
  • RNA
  • DNA