Novel degradable oligoethylenimine acrylate ester-based pseudodendrimers for in vitro and in vivo gene transfer

Gene Ther. 2008 Jan;15(1):18-29. doi: 10.1038/sj.gt.3303046. Epub 2007 Oct 25.

Abstract

A novel class of cationic hyperbranched polymers, containing branched oligoethylenimine (OEI 800 Da) as core, diacrylate esters as linkers and oligoamines as surface modification, was synthesized and evaluated regarding their structure-activity relationship as gene carriers. We show that pseudodendritic core characteristics as well as different surface modifications on the core influence DNA-binding ability, cytotoxicity and transfection efficiency. As most promising gene carrier, the pseudodendrimer HD O, that is, the OEI 800 Da core modified with hexane-1,6-diol diacrylate and surface-modified with OEI 800 Da, was identified. HD O exhibits efficient DNA-condensing ability to nanosized polyplexes (100-200 nm), low cytotoxicity, a degradation half-life of 3 days at 37 degrees C at physiological pH and in vitro reporter gene-expression levels similar to high molecular weight linear and branched polyethylenimines (PEIs) (LPEI and BPEI). In vivo studies in mice reveal that HD O/DNA polyplexes upon i.v. tail-vein injection have the potential for transfection of tumor tissue at levels comparable to that obtained with LPEI. Importantly, HD O was better tolerated than LPEI, while transgene expression was more tumor-specific and much lower in all other investigated organs, especially in the lung (15,000-fold lower compared with LPEI).

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acrylates
  • Animals
  • Aziridines
  • Cells, Cultured
  • Dendrimers / chemical synthesis*
  • Dendrimers / chemistry
  • Dendrimers / pharmacokinetics
  • Esters
  • Female
  • Genetic Engineering
  • Genetic Therapy / methods*
  • Liver / enzymology
  • Luciferases / analysis
  • Luciferases / genetics
  • Male
  • Mice
  • Mice, Inbred Strains
  • Neoplasm Transplantation
  • Neoplasms / enzymology
  • Neoplasms / therapy*
  • Polymers
  • Transfection / methods*

Substances

  • Acrylates
  • Aziridines
  • Dendrimers
  • Esters
  • Polymers
  • Luciferases