Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo

Nano Lett. 2016 Feb 10;16(2):842-8. doi: 10.1021/acs.nanolett.5b02428. Epub 2016 Jan 13.

Abstract

Safe and effective delivery is required for siRNA and mRNA-based therapeutics to reach their potential. Here, we report on the development of poly(glycoamidoamine) brush nanoparticles as delivery vehicles for siRNA and mRNA. These polymers were capable of significant delivery of siRNA against FVII and mRNA-encoding erythropoietin (EPO) in mice. Importantly, these nanoparticles were well-tolerated at their effective dose based on analysis of tissue histology, systemic cytokine levels, and liver enzyme chemistry. The polymer brush nanoparticles reported here are promising for therapeutic applications.

Keywords: erythropoietin; mRNA; nanomaterial; polymer brush; siRNA.

Publication types

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

MeSH terms

  • Animals
  • Erythropoietin / antagonists & inhibitors
  • Erythropoietin / genetics
  • Factor VII / genetics
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Humans
  • Mice
  • Nanoparticles / administration & dosage*
  • Nanoparticles / adverse effects
  • RNA, Messenger / administration & dosage*
  • RNA, Small Interfering / administration & dosage

Substances

  • RNA, Messenger
  • RNA, Small Interfering
  • Erythropoietin
  • Factor VII