Poly(beta-amino ester)s as gene delivery vehicles: challenges and opportunities

Expert Opin Drug Deliv. 2020 Oct;17(10):1395-1410. doi: 10.1080/17425247.2020.1796628. Epub 2020 Jul 31.

Abstract

Introduction: Gene delivery technologies are being developed for an increasing number of biomedical applications, with delivery vehicles including viruses and non-viral materials. Among biomaterials used for non-viral gene delivery, poly(beta-amino ester)s (PBAEs), a class of synthetic, biodegradable polymers, have risen as a leading gene delivery vehicle that has been used for multiple applications in vitro and in vivo.

Areas covered: This review summarizes the key properties of PBAEs and their development, including a discussion of the advantages and disadvantages of PBAEs for gene delivery applications. The use of PBAEs to improve the properties of other drug delivery vehicles is also summarized.

Expert opinion: PBAEs are designed to have multiple characteristics that are ideal for gene delivery, including their reversible positive charge, which promotes binding to nucleic acids as well as imparting high buffering capacity, and their rapid degradability under mild conditions. Simultaneously, some of their properties also lead to nanoparticle instability and low transfection efficiency in physiological environments. The ease with which PBAEs can be chemically modified as well as non-covalently blended with other materials, however, allows them to be customized specifically to overcome delivery barriers for varied applications.

Keywords: Biomaterials; gene delivery; nanoparticles; nucleic acids.

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

  • Biocompatible Materials / chemistry
  • Drug Delivery Systems
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Humans
  • Nanoparticles / chemistry
  • Polymers / chemistry*
  • Transfection

Substances

  • Biocompatible Materials
  • Polymers
  • poly(beta-amino ester)