A degradable polyethylenimine derivative with low toxicity for highly efficient gene delivery

M. Laird Forrest, James T. Koerber, Daniel W. Pack

Research output: Contribution to journalArticlepeer-review

368 Scopus citations

Abstract

Routine clinical implementation of human gene therapy awaits safe and efficient gene delivery methods. Polymeric vectors hold promise due to the availability of diverse chemistries, potentially providing targeting, low immunogenicity, nontoxicity, and robustness, but lack sufficient gene delivery efficiency. We have synthesized a versatile group of degradable polycations, through addition of 800-Da polyethylenimine (PEI) to small diacrylate cross-linkers. The degradable polymers reported here are similar in structure, size (14-30 kDa), and DNA-binding properties to commercially available 25-kDa PEI, but mediate gene expression two- to 16-fold more efficiently and are essentially nontoxic. These easily synthesized polymers are some of the most efficient polymeric vectors reported to date and provide a versatile platform for investigation of the effects of polymer structure and degradation rate on gene delivery efficiency.

Original languageEnglish
Pages (from-to)934-940
Number of pages7
JournalBioconjugate Chemistry
Volume14
Issue number5
DOIs
StatePublished - 2003

ASJC Scopus subject areas

  • Biotechnology
  • Bioengineering
  • Biomedical Engineering
  • Pharmacology
  • Pharmaceutical Science
  • Organic Chemistry

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