GDNF gene delivery via a 2-(dimethylamino)ethyl methacrylate based cyclized knot polymer for neuronal cell applications

ACS Chem Neurosci. 2013 Apr 17;4(4):540-6. doi: 10.1021/cn4000023. Epub 2013 Feb 11.

Abstract

Nonviral genetic therapeutic intervention strategies for neurological disorders hold great promise, but a lack of vector efficacy, coupled with vector toxicity, continue to hinder progress. Here we report the application of a newly developed class of polymer, distinctly different from conventional branched polymers, as a transfection agent for the delivery of glial cell line derived neurotrophic factor (GDNF) encoding gene. This new 2-(dimethylamino)ethyl methacrylate (DMAEMA) based cyclized knot polymer was studied for neuronal cell transfection applications, in comparison to branched polyethyleneimine (PEI). While showing a similar transfection profile over multiple cell types, the cyclized knot polymer showed far lower toxicity. In addition, transfection of Neu7 astrocytes with the GDNF encoding gene was able to cause neurite outgrowth when cocultured with dorsal root ganglia (DRGs). The cyclized knot polymer assessed here (PD-E 8%PEG), synthesized via a simple one-pot reaction, was shown to have great potential for neuronal gene therapy applications.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cells, Cultured
  • Coculture Techniques
  • Gene Transfer Techniques*
  • Glial Cell Line-Derived Neurotrophic Factor / administration & dosage*
  • Glial Cell Line-Derived Neurotrophic Factor / genetics*
  • Humans
  • Methacrylates / administration & dosage*
  • Neurons / drug effects*
  • Neurons / physiology
  • Polymers / administration & dosage*

Substances

  • GDNF protein, human
  • Glial Cell Line-Derived Neurotrophic Factor
  • Methacrylates
  • Polymers
  • 2-(dimethylamino)ethyl methacrylate