Polymer-based gene delivery with low cytotoxicity by a unique balance of side-chain termini

Proc Natl Acad Sci U S A. 2001 Jan 30;98(3):1200-5. doi: 10.1073/pnas.98.3.1200. Epub 2001 Jan 23.

Abstract

Protein expression after delivery of plasmid DNA to the cell nucleus depends on the processes of transcription and translation. Cytotoxic gene-delivery systems may compromise these processes and limit protein expression. This situation is perhaps most prevalent in current nonviral polycationic gene-delivery systems in which the polycationic nature of the delivery system can lead to cytotoxicity. To approach the problem of creating nontoxic but effective gene-delivery systems, we hypothesized that by optimizing the balance between polymer cationic density with endosomal escape moieties, effective gene transfer with low cytotoxicity could be created. As a model system, we synthesized a series of polymers whose side-chain termini varied with respect to the balance of cationic centers and endosomal escape moieties. Specifically, by polymer-analogous amidation we conjugated imidazole groups to the epsilon-amines of polylysine in varying mole ratios (73.5 mol % imidazole, 82.5 mol % imidazole, and 86.5 mol % imidazole). The primary epsilon-amine terminus of polylysine served as a model for the cationic centers, whereas the imidazole groups served as a model for the endosomal escape moieties. These polymers condensed plasmid DNA into nanostructures <150 nm and possessed little cytotoxicity in vitro. Transfection efficiency, as measured by luciferase protein expression, increased with increasing imidazole content of the polymers in a nonlinear relationship. The polymer with the highest imidazole content (86.5 mol %) mediated the highest protein expression, with levels equal to those mediated by polyethylenimine, but with little to no cytotoxicity.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Biocompatible Materials
  • Cell Line
  • Cell Survival
  • Cytomegalovirus
  • Drug Carriers
  • Gene Transfer Techniques*
  • Genes, Reporter
  • Genetic Vectors
  • Imidazoles
  • Indicators and Reagents
  • Luciferases / analysis
  • Luciferases / genetics*
  • Macrophages
  • Mice
  • Plasmids*
  • Polylysine*
  • Structure-Activity Relationship
  • Transfection / methods*

Substances

  • Biocompatible Materials
  • Drug Carriers
  • Imidazoles
  • Indicators and Reagents
  • Polylysine
  • Luciferases