Aerosol delivery of programmed cell death protein 4 using polysorbitol-based gene delivery system for lung cancer therapy

J Drug Target. 2014 Nov;22(9):829-38. doi: 10.3109/1061186X.2014.932796. Epub 2014 Jul 1.

Abstract

The development of a safe and effective gene delivery system is the most challenging obstacle to the broad application of gene therapy in the clinic. In this study, we report the development of a polysorbitol-based gene delivery system as an alternative gene carrier for lung cancer therapy. The copolymer was prepared by a Michael addition reaction between sorbitol diacrylate (SD) and spermine (SPE); the SD-SPE copolymer effectively condenses with DNA on the nanoscale and protects it from nucleases. SD-SPE/DNA complexes showed excellent transfection with low toxicity both in vitro and in vivo, and aerosol delivery of SD-SPE complexes with programmed cell death protein 4 DNA significantly suppressed lung tumorigenesis in K-ras(LA1) lung cancer model mice. These results demonstrate that SD-SPE has great potential as a gene delivery system based on its excellent biocompatibility and high gene delivery efficiency for lung cancer gene therapy.

Keywords: Accelerated transfection; aerosol delivery; gene therapy; non-viral vector; polysorbitol; polyspermine.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aerosols / chemistry*
  • Animals
  • Apoptosis Regulatory Proteins / administration & dosage*
  • Apoptosis Regulatory Proteins / pharmacology*
  • Cell Line, Tumor
  • Cell Survival
  • Disease Models, Animal
  • Gene Transfer Techniques*
  • Humans
  • Lung Neoplasms / drug therapy*
  • Mice
  • Mice, Inbred BALB C
  • RNA-Binding Proteins / administration & dosage*
  • RNA-Binding Proteins / pharmacology*
  • Sorbitol / chemistry
  • Spermine / chemistry
  • Transfection / methods

Substances

  • Aerosols
  • Apoptosis Regulatory Proteins
  • Pdcd4 protein, mouse
  • RNA-Binding Proteins
  • Spermine
  • Sorbitol