PEG-b-PPA/DNA micelles improve transgene expression in rat liver through intrabiliary infusion

J Control Release. 2007 Oct 8;122(3):297-304. doi: 10.1016/j.jconrel.2007.06.014. Epub 2007 Jun 22.

Abstract

We have developed a new block copolymer gene carrier that comprises of a polyethylene glycol segment and a degradable cationic polyphosphoramidate (PPA) segment. This PEG-b-PPA copolymer carrier formed micelles upon condensation with plasmid DNA in aqueous solution. PEG-b-PPA/DNA micelles exhibited uniform and reduced particle size ranging from 80 to 100 nm and lowered surface charge, compared with complexes of DNA with the corresponding cationic PPA carrier. PEG-b-PPA/DNA micelles maintained similar transfection efficiency as PPA/DNA complexes, which was comparable to that of PEI/DNA complexes in HepG2 cells, but yielded about 16-fold lower transgene expression in primary rat hepatocytes than PPA/DNA complexes. Following bile duct infusion in Wistar rats, PEG-b-PPA/DNA micelles mediated 4-fold higher and more uniform gene expression in the liver than PPA/DNA complexes. Liver function tests and histopathological examination indicated that PEG-b-PPA/DNA micelles showed low toxicity and good biocompatibility in the liver. This study demonstrated the potential of PEG-b-PPA/DNA micelles as an efficient carrier for liver-targeted gene delivery.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Bile Ducts
  • Biocompatible Materials* / adverse effects
  • Biocompatible Materials* / chemical synthesis
  • Biocompatible Materials* / chemistry
  • Cell Line, Tumor
  • DNA / administration & dosage*
  • DNA / genetics
  • Drug Carriers* / adverse effects
  • Drug Carriers* / chemical synthesis
  • Drug Carriers* / chemistry
  • Gene Targeting / methods*
  • Hepatocytes / drug effects
  • Hepatocytes / metabolism
  • Humans
  • Infusion Pumps
  • Liver / drug effects*
  • Liver / metabolism
  • Micelles
  • Microscopy, Electron, Transmission
  • Organophosphates / chemical synthesis
  • Organophosphates / chemistry
  • Organophosphates / metabolism
  • Particle Size
  • Polyethylene Glycols / chemical synthesis
  • Polyethylene Glycols / chemistry
  • Polyethylene Glycols / metabolism
  • Rats
  • Transfection
  • Transgenes* / genetics

Substances

  • Biocompatible Materials
  • Drug Carriers
  • Micelles
  • Organophosphates
  • Polyethylene Glycols
  • DNA