Metabolically stabilized long-circulating PEGylated polyacridine peptide polyplexes mediate hydrodynamically stimulated gene expression in liver

Gene Ther. 2011 Jan;18(1):23-37. doi: 10.1038/gt.2010.117. Epub 2010 Aug 19.

Abstract

A novel class of PEGylated polyacridine peptides was developed that mediate potent stimulated gene transfer in the liver of mice. Polyacridine peptides, (Acr-X)(n)-Cys-polyethylene glycol (PEG), possessing 2-6 repeats of Lys-acridine (Acr) spaced by either Lys, Arg, Leu or Glu, were Cys derivatized with PEG (PEG(5000 kDa)) and evaluated as in vivo gene transfer agents. An optimal peptide of (Acr-Lys)(6)-Cys-PEG was able to bind to plasmid DNA (pGL3) with high affinity by polyintercalation, stabilize DNA from metabolism by DNAse and extend the pharmacokinetic half-life of DNA in the circulation for up to 2 h. A tail vein dose of PEGylated polyacridine peptide pGL3 polyplexes (1 μg in 50 μl), followed by a stimulatory hydrodynamic dose of normal saline at times ranging from 5 to 60 min post-DNA administration, led to a high level of luciferase expression in the liver, equivalent to levels mediated by direct hydrodynamic dosing of 1 μg of pGL3. The results establish the unique properties of PEGylated polyacridine peptides as a new and promising class of gene delivery peptides that facilitate reversible binding to plasmid DNA, protecting it from DNase in vivo resulting in an extended circulatory half-life, and release of transfection-competent DNA into the liver to mediate a high-level of gene expression upon hydrodynamic boost.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Acridines / chemistry
  • Acridines / metabolism*
  • Animals
  • DNA / administration & dosage*
  • DNA / chemistry
  • DNA / metabolism
  • Gene Expression*
  • Gene Transfer Techniques
  • Half-Life
  • Hydrodynamics
  • Liver / metabolism*
  • Mice
  • Peptides / chemistry
  • Peptides / metabolism
  • Plasmids / genetics
  • Plasmids / metabolism
  • Polyethylene Glycols / chemistry
  • Polyethylene Glycols / metabolism*
  • Transfection

Substances

  • Acridines
  • Peptides
  • Polyethylene Glycols
  • DNA